KSQ Therapeutics Announces FDA Clearance of IND Application for KSQ-004EX, a CRISPR/Cas9 Engineered Tumor-Infiltrating Lymphocyte (eTIL®) Therapy

Phase 1/2 clinical study will evaluate the safety and preliminary efficacy of KSQ-004EX in advanced solid tumors

LEXINGTON, Mass., Sept. 24, 2024 (GLOBE NEWSWIRE) — KSQ Therapeutics, Inc. (KSQ), a clinical-stage biotechnology company developing novel treatments for solid tumors, in collaboration with The University of Texas MD Anderson Cancer Center (MD Anderson) and the Cell Therapy Manufacturing Center (CTMC), today announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a Phase 1/2 clinical study of KSQ-004EX, KSQ’s second engineered tumor-infiltrating lymphocyte (eTIL^®) program. KSQ-004EX consists of TIL in which the genes encoding SOCS1 and Regnase-1 are inactivated using CRISPR/Cas9 gene editing. KSQ’s CRISPRomics^® platform identified SOCS1 and Regnase-1 as key regulators of anti-tumor potency and persistence of TIL in preclinical models, providing KSQ-004EX with best-in-class potential in the treatment of advanced solid tumor indications.

“As we continue to learn more about the biology of our eTIL candidates, we gain confidence that they can redefine the treatment of solid tumors, including difficult-to-treat cancers. We’re excited to bring an additional therapy into the clinic with the experts at MD Anderson, where this study will be initiated,” said Qasim Rizvi, Chief Executive Officer of KSQ.

“Solid tumor treatment continues to be a significant challenge in oncology. The ability to offer patients a novel cell therapy is encouraging,” said Rodabe Amaria, M.D., professor of Melanoma Medical Oncology at MD Anderson and principal investigator of the KSQ-004EX study. “The preclinical KSQ-004EX data is promising, and we look forward to exploring the potential of KSQ’s dual-edited TIL.”

Phase 1/2 Trial Design

The Phase 1/2 clinical trial is an open-label, dose-escalation study for patients with select advanced solid tumors. The primary objective of the Phase 1 portion of the trial is to evaluate the safety and tolerability of KSQ-004EX. The primary objective of Phase 2 is to evaluate anti-tumor activity in multiple indication-specific cohorts.

About KSQ-004EX

KSQ-004EX, an eTIL^® cell therapy in which TIL are edited to inactivate the genes encoding SOCS1 and Regnase-1, has the potential to revolutionize the treatment of solid tumors. In preclinical studies, KSQ-004EX demonstrated enhanced anti-tumor function in solid tumor models refractory to PD-1 inhibition. KSQ-004EX is manufactured by CTMC using KSQ’s ExPRESS^™ process using patient tumor samples, including core biopsies, as starting material.

About KSQ Therapeutics

KSQ Therapeutics is advancing a pipeline of tumor- and immune-focused drug candidates to treat cancer across multiple drug modalities, including targeted therapies, adoptive cell therapies, and immunotherapies. KSQ’s proprietary CRISPRomics^® discovery engine enables genome-scale, in vivo validated, unbiased target discovery across broad therapeutic areas. For more information, please visit www.ksqtx.com and follow us on LinkedIn and X.

KSQ Media Contact:

Cory Tromblee
cory@scientpr.com