September 17, 2021
1 min read
The FDA cleared an investigational new drug application for NTLA-5001, a T-cell receptor T-cell therapy designed to treat adults with relapsed or refractory acute myeloid leukemia.
NTLA-5001 (Intellia Therapeutics) is an autologous, gene-edited T-cell receptor (TCR) T-cell therapy. The agent uses ex vivo CRISPR gene editing to insert TCRs on a patient’s T cells that target the Wilms’ tumor 1 (WT1) antigen on the surface of cancer cells.
“The FDA’s acceptance of our IND for NTLA-5001 is an important milestone in our pursuit of developing advanced cell therapies utilizing Intellia’s proprietary engineering platform to treat patients with cancer,” John Leonard, MD, president and CEO of Intellia, said in a company-issued press release.
“Our treatment strategy is to leverage CRISPR/Cas9 genome editing technology to create next-generation engineered immune cells with the potential to attack cancer cells more effectively and safely than previously developed cell therapies,” he added. “Our study is an important first step toward improving treatment for people living with this aggressive form of cancer.”
The IND clearance will allow Intellia to begin enrollment of a trial designed to evaluate NTLA-5001 for adults with relapsed or refractory AML who previously received standard first-line therapy. The study — designed to enroll up to 54 patients — will include both dose-escalation and dose-expansion phases.
The multi-arm phase 1/phase 2A trial will evaluate the safety, tolerability, cell kinetics and antitumor activity of a single dose of NTLA-5001. The dose-escalation phase will include two independent arms of up to three cohorts based on the patients’ levels of disease burden.
Enrollment is scheduled to begin by the end of the year.
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