Trial Outcomes & Findings for A Study of Ad-RTS-hIL-12 With Veledimex in Combination With Nivolumab in Subjects With Glioblastoma; a Substudy to ATI001-102 (NCT NCT03636477)

Last Updated: 2025-08-12

Results Overview

Evaluation of adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v4.03 will be based on the incidence, intensity and type of adverse event. AEs will be regarded as treatment-emergent adverse events (TEAEs) during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

21 participants

Primary outcome timeframe

2 years and 4 months

Results posted on

2025-08-12

Participant Flow

Subject enrollment and dose escalation followed a 3+3 design. Each subject in the 1st cohort, Cohort S1, was monitored through Day 28 before next subject was dosed. The first subjects in the subsequent cohorts, Cohorts S2 and S3, were monitored through Day 28. After completion of Cohort S3, the Safety Review Committee (SRC) and Data Safety Monitoring Board (DSMB )recommended expanding accrual, and additional 12 subjects were enrolled in an expansion cohort (20mg veledimex and 3mg/kg nivolumab).

Participant milestones

Participant milestones
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg once daily (QD) on Days 0-14 Nivolumab 1 mg/kg intravenously (IV) over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Overall Study STARTED	3	3	15
Overall Study COMPLETED	3	3	14
Overall Study NOT COMPLETED	0	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg once daily (QD) on Days 0-14 Nivolumab 1 mg/kg intravenously (IV) over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Overall Study Adverse Event	0	0	1

Baseline Characteristics

A Study of Ad-RTS-hIL-12 With Veledimex in Combination With Nivolumab in Subjects With Glioblastoma; a Substudy to ATI001-102

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Total n=21 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants	13 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	1 Participants n=7 Participants	7 Participants n=5 Participants	8 Participants n=4 Participants
Age, Continuous	42.90 years STANDARD_DEVIATION 17.34 • n=5 Participants	59.37 years STANDARD_DEVIATION 7.21 • n=7 Participants	59.75 years STANDARD_DEVIATION 13.54 • n=5 Participants	57.29 years STANDARD_DEVIATION 14.14 • n=4 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	7 Participants n=5 Participants	9 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants	12 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants	20 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants	20 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Height	178.67 cm STANDARD_DEVIATION 8.93 • n=5 Participants	175.27 cm STANDARD_DEVIATION 14.17 • n=7 Participants	147.94 cm STANDARD_DEVIATION 10.15 • n=5 Participants	175.52 cm STANDARD_DEVIATION 10.10 • n=4 Participants
Weight	74.33 kg STANDARD_DEVIATION 11.62 • n=5 Participants	82.83 kg STANDARD_DEVIATION 29.11 • n=7 Participants	85.19 kg STANDARD_DEVIATION 19.84 • n=5 Participants	83.30 kg STANDARD_DEVIATION 19.71 • n=4 Participants
Disease Status at Entry Unifocal	2 Participants n=5 Participants	3 Participants n=7 Participants	10 Participants n=5 Participants	15 Participants n=4 Participants
Disease Status at Entry Multifocal	1 Participants n=5 Participants	0 Participants n=7 Participants	5 Participants n=5 Participants	6 Participants n=4 Participants
Disease Status at Entry Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Number of Recurrences 1st recurrence	2 Participants n=5 Participants	3 Participants n=7 Participants	10 Participants n=5 Participants	15 Participants n=4 Participants
Number of Recurrences >=2 recurrence	1 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants	5 Participants n=4 Participants
Number of Recurrences Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants
Isocitrate Dehydrogenase (IDH) Mutation Status at Diagnosis Mutated	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants
Isocitrate Dehydrogenase (IDH) Mutation Status at Diagnosis Wild-Type	2 Participants n=5 Participants	3 Participants n=7 Participants	14 Participants n=5 Participants	19 Participants n=4 Participants
Isocitrate Dehydrogenase (IDH) Mutation Status at Diagnosis Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
MGMT Status at Diagnosis Methylated	2 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants	8 Participants n=4 Participants
MGMT Status at Diagnosis Unmethylated	1 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants	11 Participants n=4 Participants
MGMT Status at Diagnosis Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	2 Participants n=4 Participants
Karnofsky Performance Score (KPS) at Screening >= 70-90	0 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants	4 Participants n=4 Participants
Karnofsky Performance Score (KPS) at Screening >= 90	3 Participants n=5 Participants	3 Participants n=7 Participants	11 Participants n=5 Participants	17 Participants n=4 Participants
Karnofsky Performance Score (KPS) at Screening Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Prior Lines of Treatment Greater Than One	0 Participants n=5 Participants	0 Participants n=7 Participants	5 Participants n=5 Participants	5 Participants n=4 Participants
Prior Lines of Treatment One	3 Participants n=5 Participants	3 Participants n=7 Participants	10 Participants n=5 Participants	16 Participants n=4 Participants
Prior Steroid Use Yes	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	3 Participants n=4 Participants
Prior Steroid Use No	3 Participants n=5 Participants	3 Participants n=7 Participants	12 Participants n=5 Participants	18 Participants n=4 Participants
Prior Steroid Use Missing	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

PRIMARY outcome

Timeframe: 2 years and 4 months

Population: The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Number of Participants With Adverse Events (AEs) Any TEAEs	3 participants	3 participants	15 participants
Number of Participants With Adverse Events (AEs) Any Serious TEAEs	1 participants	2 participants	6 participants
Number of Participants With Adverse Events (AEs) Any TEAEs with Toxicity Grade ≥ 3	3 participants	3 participants	11 participants
Number of Participants With Adverse Events (AEs) Any TEAEs Leading to Treatment Dose Modification	2 participants	2 participants	4 participants
Number of Participants With Adverse Events (AEs) Any TEAEs Leading to Treatment Discontinuations	0 participants	2 participants	2 participants
Number of Participants With Adverse Events (AEs) Any TEAEs Leading to Death	0 participants	0 participants	1 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related TEAEs	3 participants	3 participants	15 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related Serious TEAEs	1 participants	1 participants	3 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related TEAEs with Toxicity Grade ≥ 3	2 participants	3 participants	7 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related TEAEs Leading to Treatment Dose Modification	2 participants	1 participants	4 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related TEAEs Leading to Treatment Discontinuations	0 participants	1 participants	2 participants
Number of Participants With Adverse Events (AEs) Any Drug-Related TEAEs Leading to Death	0 participants	0 participants	0 participants

PRIMARY outcome

Timeframe: From Day 0 through Day 14 for each participant

Evaluation will be based on expected dose compliance. Subjects were instructed to document veledimex dosing compliance in a subject diary, including the time each dose was taken, the time of the last meal prior to administration of veledimex, the number of capsules taken, whether the subject missed any veledimex doses, and reason for any missed doses. Investigational product container(s) with any remaining capsules were returned to the study staff on Day 15, and staff assessed dose compliance.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Number of Participants With Veledimex Dose Compliance 100% compliance	2 participants	3 participants	11 participants
Number of Participants With Veledimex Dose Compliance Less than 100%	1 participants	0 participants	4 participants
Number of Participants With Veledimex Dose Compliance 80% or more	3 participants	3 participants	15 participants
Number of Participants With Veledimex Dose Compliance Less than 80%	0 participants	0 participants	0 participants

SECONDARY outcome

Timeframe: The first treatment cycle (21 days).

The primary objective was to determine the Maximum Tolerated Dose (MTD), defined as the dose at which fewer than 33% of subjects experience a Dose-Limiting Toxicity (DLT). The MTD was not reached; a Maximum Administered Dose (MAD) of 20mg veledimex and 3mg/kg nivolumab was determined. This measure reports the number of subjects who experienced a DLT during the first treatment cycle in each dose cohort.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Number of Participants With a Dose-Limiting Toxicity (DLT)	0 participants	1 participants	2 participants

SECONDARY outcome

Timeframe: 2 years and 4 months

Population: The Evaluable Safety Population (ESP) includes subjects who have received Ad-RTS-hIL-12 and at least one dose of veledimex after Ad-RTS-hIL-12 administration. The ESP will be evaluated for investigator assessment of objective response rate (ORR).

To determine investigator assessment of response including tumor ORR of Ad-RTS-hIL-12 + veledimex when administered in combination with nivolumab. Investigator assessment of ORR was determined according to Immunotherapy Response Assessment in Neuro-Oncology (iRANO) criteria. iRANO is a set of criteria built on RANO criteria but adapts them for immune-related effects to evaluate treatment response in brain tumor patients receiving immunotherapy. It addresses unique challenges like pseudoprogression, where immune-related inflammation mimics tumor growth on imaging allowing continued treatment despite early radiographic worsening if the patient is clinically stable. This criteria requires confirmation of progression with follow-up imaging ≥3 months later, especially within the first 6 months of immunotherapy.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Tumor Objective Response Rate (ORR) Complete Response	0 Participants	0 Participants	0 Participants
Tumor Objective Response Rate (ORR) Partial Response	0 Participants	1 Participants	0 Participants
Tumor Objective Response Rate (ORR) Stable Disease	2 Participants	1 Participants	11 Participants
Tumor Objective Response Rate (ORR) Progressive Disease	1 Participants	1 Participants	4 Participants
Tumor Objective Response Rate (ORR) Not Evaluable	0 Participants	0 Participants	0 Participants
Tumor Objective Response Rate (ORR) No Response	0 Participants	0 Participants	0 Participants
Tumor Objective Response Rate (ORR) Missing	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years and 4 months

PFS is the time in days from the first treatment (either veledimex or Ad-RTS-hIL-12) to the first assessment on which the overall response is reported as disease progression (per Response Assessment in Neuro-Oncology \[RANO\] or Immunotherapy Response Assessment in Neuro-Oncology \[iRANO\] criteria). Subjects withdrawing from the study will be censored at their last non progressive disease response assessment. If a subject does not have a non-progressive disease response assessment, the subject will be censored on the date of the first treatment as described above.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Progression Free Survival (PFS)	38.0 Days Standard Deviation 41.6	88.0 Days Standard Deviation 98.8	36.9 Days Standard Deviation 29.0

SECONDARY outcome

Timeframe: 2 years and 4 months

Population: The Evaluable Safety Population (ESP) includes subjects who have received Ad-RTS-hIL-12 and at least one dose of veledimex after Ad-RTS-hIL-12 administration. The ESP is denoted as the PP population in this uncontrolled setting.

PSP -- Progression free survival was originally defined for determination of PSP requiring confirmation of progression (per Response Assessment in Neuro-Oncology \[RANO\] or Immunotherapy Response Assessment in Neuro-Oncology \[iRANO\] criteria).

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Rate of Pseudo-progression (PSP)	0 participants	1 participants	0 participants

SECONDARY outcome

Timeframe: Up to 24 months

Population: The Overall Safety Population (OSP) included all subjects who received at least one dose of any study drug.

The percentage of participants alive at each time point (6, 9, 12, 15, 18, and 24 months) are reported.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Overall Survival (OS) OS at Month 24	0 Percentage of participants	33.3 Percentage of participants	0 Percentage of participants
Overall Survival (OS) OS at Month 6	100 Percentage of participants	66.7 Percentage of participants	60.0 Percentage of participants
Overall Survival (OS) OS at Month 9	66.7 Percentage of participants	66.7 Percentage of participants	46.7 Percentage of participants
Overall Survival (OS) OS at Month 12	33.3 Percentage of participants	66.7 Percentage of participants	40 Percentage of participants
Overall Survival (OS) OS at Month 15	33.3 Percentage of participants	66.7 Percentage of participants	26.7 Percentage of participants
Overall Survival (OS) OS at Month 18	33.3 Percentage of participants	66.7 Percentage of participants	13.3 Percentage of participants

SECONDARY outcome

Timeframe: From Screening through Day 28, assessed at Screening and Days 0, 1, 3, 7, 14, and 28

Population: Evaluable Safety Population (ESP) includes subjects who have received nivolumab, Ad-RTS-hIL-12, and at least one dose of veledimex. Pharmacodynamic Population (PDP) refers to ESP.

Blood samples for pharmacodynamic biomarker evaluation were collected at screening, during treatment, and post-treatment. The immunological and biological response markers include serum cytokines (IL-12 and IFNℽ), and T and B cell subpopulations. Serum IL-12 and downstream IFNℽ expressions are reported by time point.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Screening	1.19 pg/mL Standard Deviation 0.20	0.73 pg/mL Standard Deviation 0.15	0.40 pg/mL Standard Deviation 0.32
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 0	0.93 pg/mL Standard Deviation 0.26	1.12 pg/mL Standard Deviation 0.69	0.64 pg/mL Standard Deviation 0.34
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 1	2.16 pg/mL Standard Deviation 2.03	0.90 pg/mL Standard Deviation 0.36	0.82 pg/mL Standard Deviation 0.38
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 3	6.05 pg/mL Standard Deviation 3.70	2.22 pg/mL Standard Deviation 1.34	9.00 pg/mL Standard Deviation 11.57
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 7	1.98 pg/mL Standard Deviation 0	3.84 pg/mL Standard Deviation 0.42	4.32 pg/mL Standard Deviation 3.82
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 14	5.41 pg/mL Standard Deviation 4.75	1.01 pg/mL Standard Deviation 0.29	2.58 pg/mL Standard Deviation 2.49
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IL12 Day 28	1.32 pg/mL Standard Deviation 0.50	0.84 pg/mL Standard Deviation 0.23	0.64 pg/mL Standard Deviation 0.33
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Screening	8.93 pg/mL Standard Deviation 1.62	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 0	8.1 pg/mL Standard Deviation 0.17	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 1	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 3	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	2.12 pg/mL Standard Deviation 5.40
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 7	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	5.46 pg/mL Standard Deviation 7.22
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 14	13.9 pg/mL Standard Deviation 8.34	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	7.68 pg/mL Standard Deviation 28.73
Changes From Baseline in Cellular Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. Serum IFN gamma Day 28	NA pg/mL Standard Deviation NA All values were below the limit of quantitation (BLQ) of 8 pg/mL.	8.70 pg/mL Standard Deviation 1.21	3.52 pg/mL Standard Deviation 12.67

SECONDARY outcome

Timeframe: From Screening through Day 28, assessed at Screening, Days 0, 14, and 28

Population: Evaluable Safety Population (ESP) includes subjects who have received nivolumab, Ad-RTS-hIL-12, and at least one dose of veledimex. Pharmacodynamic Population (PDP) refers to ESP.

Blood samples for pharmacodynamic biomarker evaluation were collected at screening, during treatment, and post-treatment. Whole blood flow cytometry was used to assess the circulating blood cell subpopulations (e.g., T-reg and T cell panels).

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD45+ [pan-T] at Screening	62 percentage Standard Deviation 15.56	78 percentage Standard Deviation 7.07	75 percentage Standard Deviation 14.14
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD45+ [pan-T] at Day 0	58.67 percentage Standard Deviation 8.39	74.33 percentage Standard Deviation 7.51	75.5 percentage Standard Deviation 3.54
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD45+ [pan-T] at Day 14	71.33 percentage Standard Deviation 7.37	73.67 percentage Standard Deviation 9.08	78 percentage Standard Deviation 3.83
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD45+ [pan-T] at Day 28	69 percentage Standard Deviation 7.55	77.33 percentage Standard Deviation 9.29	73 percentage Standard Deviation 2.83
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD8+ [Tcytotoxic] at Screening	21 percentage Standard Deviation 2.83	30.5 percentage Standard Deviation 0.71	32.5 percentage Standard Deviation 2.12
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD8+ [Tcytotoxic] at Day 0	24 percentage Standard Deviation 3.61	31 percentage Standard Deviation 5	30 percentage Standard Deviation 4.24
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD8+ [Tcytotoxic] at Day 14	31.67 percentage Standard Deviation 9.50	30.33 percentage Standard Deviation 1.15	29.5 percentage Standard Deviation 6.36
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+ CD8+ [Tcytotoxic] at Day 28	29 percentage Standard Deviation 10.15	30.67 percentage Standard Deviation 4.62	31 percentage Standard Deviation 0
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+/ CD4+/ CD25hi/ Foxp3+/ CD127lo- [Treg] at Screening	1.1 percentage Standard Deviation 0.57	1.8 percentage Standard Deviation 0.99	0.35 percentage Standard Deviation 0.49
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+/ CD4+/ CD25hi/ Foxp3+/ CD127lo- [Treg] at Day 0	0.97 percentage Standard Deviation 0.38	0.85 percentage Standard Deviation 0.49	1.1 percentage Standard Deviation 0.99
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+/ CD4+/ CD25hi/ Foxp3+/ CD127lo- [Treg] at Day 14	0.8 percentage Standard Deviation 0.78	1.70 percentage Standard Deviation 1.50	0.35 percentage Standard Deviation 0.35
Changes From Baseline in Humoral Immune Responses Elicited by Ad-RTS-hIL-12 and Veledimex in Combination With Nivolumab. CD3+/ CD4+/ CD25hi/ Foxp3+/ CD127lo- [Treg] at Day 28	1.27 percentage Standard Deviation 1.24	2.9 percentage Standard Deviation 2.26	0.95 percentage Standard Deviation 0.92

SECONDARY outcome

Timeframe: Day 0 to Day 15 (Day 14 24-hour post dose)

Population: All subjects with available plasma-time concentration data are included in the PK Population.

Cmax was determined from the maximum plasma concentration from Day 0 (post veledimex and resection/craniotomy), 3-5 hours after the veledimex dose on Day 1, and 3-5 hours after the veledimex dose on Day 14.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Maximum Plasma Concentration (Cmax)	11.045 ng/mL Standard Deviation 6.159	13.707 ng/mL Standard Deviation 5.423	75.68 ng/mL Standard Deviation 82.38

SECONDARY outcome

Timeframe: Day 0 to Day 15 (Day 14 24-hour post dose)

Population: All subjects with available plasma-time concentration data are included in the PK Population.

Tmax was determined from the time of maximum plasma concentration from Day 0 (post veledimex and resection/craniotomy), 3-5 hours after the veledimex dose on Day 1, and 3-5 hours after the veledimex dose on Day 14.

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Time to Maximum Plasma Concentration (Tmax)	3.95 Hours Standard Deviation 0.12	4.00 Hours Standard Deviation 1.18	4.63 Hours Standard Deviation 2.79

SECONDARY outcome

Timeframe: Day 0 to Day 15 (24 hours post Day 14 dose)

Population: All subjects with available plasma-time concentration data are included in the PK Population.

t1/2 was determined from the plasma concentrations measured from Day 0 (post veledimex and resection/craniotomy) through Day 15 (24 hours post Day 14 dose)

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Half-life (t1/2)	1.39 hours Standard Deviation 0.14	1.79 hours Standard Deviation 0.50	1.63 hours Standard Deviation 0.77

SECONDARY outcome

Timeframe: Day 0 to Day 15 (Day 14 24-hour post dose)

Population: All Subjects with available plasma-time concentration data are included in the PK Population.

AUC was determined from the plasma concentrations measured from Day 0 (post veledimex and resection/craniotomy) through Day 15 (24 hours post Day 14 dose)

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Area Under the Concentration-versus-time Curve (AUC)	24.85 ng*hr/mL Standard Deviation 13.82	30.76 ng*hr/mL Standard Deviation 5.42	185.34 ng*hr/mL Standard Deviation 215.77

SECONDARY outcome

Timeframe: Day 0 to Day 15 (Day 14 24-hour post dose)

Population: All subjects with available plasma-time concentration data are included in the PK Population.

Vd was determined from the plasma concentrations measured from Day 0 (post veledimex and resection/craniotomy) through Day 15 (24 hours post Day 14 dose).

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Volume of Distribution (Vd)	1.07 L Standard Deviation 0.60	0.83 L Standard Deviation 0.39	0.53 L Standard Deviation 0.37

SECONDARY outcome

Timeframe: Day 0 to Day 15 (Day 14 24-hour post dose)

Population: All subjects with available plasma-time concentration data are included in the PK Population.

CL was determined from the plasma concentrations measured from Day 0 (post veledimex and resection/craniotomy) through Day 15 (24 hours post Day 14 dose).

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=2 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Pharmacokinetic Profile: Clearance (CL)	0.476 L/hour Standard Deviation 0.265	0.332 L/hour Standard Deviation 0.060	0.219 L/hour Standard Deviation 0.142

SECONDARY outcome

Timeframe: 1 day (Day 0 at time of resection)

Population: All subjects with available tumor concentration data are included in the PK Population for determination of tumor/plasma concentration ratio.

Tumor/plasma ratio at Day 0 by cohort

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=2 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=14 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Veledimex Concentration Ratio Between the Brain Tumor and the Blood.	0.561 ratio Standard Deviation 0.212	0.404 ratio Standard Deviation 0.87	0.632 ratio Standard Deviation 0.446

SECONDARY outcome

Timeframe: Days 0 through 14

Population: The Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex and/or Ad-RTS-hIL-12

To assess concomitant corticosteroid use during the first treatment cycle, the cumulative dose of dexamethasone administered to each subject from Day 0 to Day 14 was calculated. This measure reports the mean cumulative dose in milligrams (mg) for each cohort

Outcome measures

Outcome measures
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 Participants Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 Participants Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 Participants Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Cumulative Dexamethasone Use During Days 0-14	82.7 mg Standard Deviation 88.6	20 mg Standard Deviation 0	60.1 mg Standard Deviation 100.2

Adverse Events

Veledimex 10mg Dose Level + Nivolumab 1mg/kg

Serious events: 1 serious events

Other events: 3 other events

Deaths: 3 deaths

Veledimex 10mg Dose Level + Nivolumab 3mg/kg

Serious events: 2 serious events

Other events: 3 other events

Deaths: 3 deaths

Veledimex 20mg Dose Level + Nivolumab 3mg/kg

Serious events: 6 serious events

Other events: 15 other events

Deaths: 15 deaths

Serious adverse events

Serious adverse events
Measure	Veledimex 10mg Dose Level + Nivolumab 1mg/kg n=3 participants at risk Cohort S1: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 1 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 10mg Dose Level + Nivolumab 3mg/kg n=3 participants at risk Cohort S2: Ad-RTS-hIL-12 on Day 0 Veledimex 10 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks	Veledimex 20mg Dose Level + Nivolumab 3mg/kg n=15 participants at risk Cohort S3: Ad-RTS-hIL-12 on Day 0 Veledimex 20 mg QD on Days 0-14 Nivolumab 3 mg/kg IV over 60 minutes on Day -7, Day 15, and approx every 2 weeks
Nervous system disorders Brain Oedema	33.3% 1/3 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	33.3% 1/3 • Number of events 2 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	13.3% 2/15 • Number of events 2 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Nervous system disorders Seizure	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Injury, poisoning and procedural complications Fall	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Injury, poisoning and procedural complications Wound Dehiscence	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Blood and lymphatic system disorders Cold Type Haemolytic Anaemia	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Immune system disorders Cytokine Release Syndrome	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Infections and infestations Urinary Tract Infection	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	6.7% 1/15 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	33.3% 1/3 • Number of events 2 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/15 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).
Immune system disorders Immune Reconstitution Inflammatory Syndrome	0.00% 0/3 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	33.3% 1/3 • Number of events 1 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).	0.00% 0/15 • 2 years and 4 months The Full Analysis Set (FAS): Overall Safety Population (OSP) includes all subjects who have received at least one dose of veledimex (pretumor resection and) and/or all subjects who received Ad-RTS-hIL-12. AEs will be regarded as treatment-emergent TEAEs during the treatment period regardless of relationship to study drug if: • The AE occurred or worsened from baseline during or after administration of the first dose of study drug (on or after Day 0).

Other adverse events

Additional Information

Jaymes Holland

Alaunos Therapeutics

Phone: 6502732627

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Disclosure shall not occur until (i) twenty-four (24) months have elapsed since Sponsor has notified Institution of the completion of the multi-center Study in order for Sponsor to comply with its obligations under 21 CFR 312.53(c)(4), or (ii) after the publication of the multi-center Study data, if the Study is a multi-center Study or Sponsor confirms in writing there will be no multi-center publication.
Publication restrictions are in place

Restriction type: OTHER