A Phase 1/2 Study of KSQ-001EX, Autologous Tumor Infiltrating Lymphocytes Engineered to Inactivate the SOCS1 Gene, in Patients With Select Advanced Solid Tumors

Description

Phase I Primary Objectives • To evaluate the safety and tolerability of KSQ-001EX in adult participants with advanced solid tumors (melanoma, HNSCC, NSCLC)

Phase I Primary Endpoint

• Incidence of dose-limiting toxicities (DLTs)

Phase I Secondary Objectives

* Determine expansion dose
* Assess the safety and tolerability of KSQ-001EX in participants with advanced solid tumors (melanoma, HNSCC, NSCLC)
* Evaluate preliminary antitumor activity of KSQ-001EX in participants with advanced solid tumors
* Evaluate the feasibility of the manufacturing process.

Phase I Secondary Endpoints

* Incidence and severity of treatment-emergent adverse events (TEAEs) and change from Baseline in laboratory results
* Objective response rate (ORR), duration of response (DOR), time to response (TTR) as assessed by the Investigator, per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
* Manufacturing success rate

Phase 2 Primary Objectives • To assess the anti-tumor activity of KSQ-001EX in patients with advanced malignant solid tumors

Phase 2 Primary Endpoint

• ORR per RECIST v1.1

Phase 2 Secondary Objectives

* Assess the safety and tolerability of KSQ-001EX in patients with advanced solid tumors (melanoma, HNSCC, NSCLC)
* Evaluate anti-tumor activity of KSQ-001EX in patients with advanced malignant solid tumors
* Evaluate overall survival (OS)
* Evaluate the feasibility of the manufacturing process

Phase 2 Secondary Endpoints

* Incidence and severity of TEAEs and change from Baseline in laboratory results
* Complete response rate (CRR), DOR, TTR, progression-free survival (PFS) per RECIST v1.1
* OS
* Manufacturing success rate

Phase 1/2 Exploratory Objectives

* Determine persistence of KSQ-001EX
* Assess changes in immune and pharmacodynamic markers following KSQ-001EX infusion
* Assess correlations of KSQ-001EX biomarkers and KSQ-001EX DP characteristics with safety, clinical activity, and KSQ-001EX persistence

Phase 1/2 Exploratory Endpoints

* KSQ-001EX levels in blood and tumor tissue
* Change from Baseline in soluble immune factors, lymphocytes and immune markers
* KSQ-001EX pharmacodynamic markers, KSQ-001EX DP release criteria, and KSQ-001EX persistence