FDA Clears IND for Tumor Infiltrating Lymphocyte Therapy


A phase 1/2 study of KSQ-001EX will commence at MD Anderson Cancer Center following this investigational new drug approval from the FDA.

FDA logo
  • An investigational new drug (IND) has been accepted by the FDA for KSQ-001EX .
  • KSQ-001Ex is an engineered tumor infiltrating lymphocyte (TIL).
  • A phase 1/2 study will begin with this approval.

The FDA has approved an IND application for a phase 1/2 study of KSQ-001EX, an engineered TIL program, according to a press release from KSQ Therapeutics.1

“New treatment options are urgently needed in melanoma and other solid tumors, and the potential to improve outcomes and patient experience is very exciting. KSQ-001EX exhibited considerable efficacy in preclinical studies,” said Rodabe Amaria, MD, professor of melanoma medical oncology at MD Anderson and principal investigator of the KSQ-001EX study, in a press release.

The phase 1/2 study will be initiated at the MD Anderson Cancer Center and is an open-label, safety-lead in study for patients with melanoma, head and neck squamous cell carcinoma, and non­–small cell lung cancer. Phase 1 will evaluate KSQ-001EX’s safety and tolerability. Patients in the safety lead-in portion will be initially dosed without interleukin-2.Phase 2 will evaluate the antitumor activity of KSQ-001EX in indication-specific cohorts.

KSQ-001EX is composed of TILs where CRISPR/Cas9 gene editing inhibits SOCS1. Investigators identified SOCS1 as a key gene that regulates TIL’s antitumor potency and persistence, as well as provides KSQ-001EX with the best potential to treat solid tumors.

In preclinical studies, KSQ-001EX showed enhanced antitumor function in PD-1 inhibition-refractory solid tumor models. It also demonstrated enhanced persistence and memory formation. Specifically, KSQ-001 manufactured from human melanoma TIL demonstrated an in vitro profile consistent with higher anti-tumor potency than TIL, plus heightened IFNg and TNFa production.2

"Through our platform insights, we've identified how to enhance the effectiveness of T cells in the treatment of solid tumors. We believe our eTIL cell therapies have tremendous potential to benefit patients with cancer who have limited treatment options. said Qasim Rizvi, chief executive officer of KSQ, in a press release.1

Several other studies on TILs in solid tumors are ongoing. A phase 2 trial (NCT03801083) evaluating the overall response rate of the adoptive transfer of TILs in biliary tract cancer is recruiting patients at the University of Pittsburgh.3 Additionally, a phase 1 study (NCT06047977) investigating the feasibility of creating TIL from high-risk pediatric solid tumors is beginning at Johns Hopkins All Children’s Hospital and will soon begin recruiting.4

1. KSQ Therapeutics announces FDA clearance of IND application for KSQ-001EX, a CRISPR/Cas9 engineereed tumor infiltrating lymphocyte (eTIL) therapy. News release. KSQ Therapeutics. November 29, 2023. Accessed November 29, 2023. https://tinyurl.com/zydmxfrb
2. Colletti N, LaMothe R, Bullock C, et al. KSQ-001: A CRISPR/Cas9-engineered tumor infiltrating lymphocyte (eTIL) therapy for solid tumors. KSQ Therapeutics. Accessed November 29, 2023. https://tinyurl.com/kta9mudb
3. Adoptive transfer of tumor infiltrating lymphocytes for biliary tract cancers. ClinicalTrials.gov. Updated June 27, 2023. Accessed November 29, 2023. https://clinicaltrials.gov/study/NCT03801083
4. Tumor infiltrating lymphocyte therapy for pediatric high risk solid tumors. ClinicalTrials.gov. Updated September 28, 2023. Accessed November 29, 2023. https://clinicaltrials.gov/study/NCT06047977
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