On March 9, 2022 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapeutics leveraging CRISPR-based technologies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia’s ex vivo investigational T cell receptor (TCR)-T cell therapy, NTLA-5001, for the treatment of acute myeloid leukemia (AML) (Press release, Intellia Therapeutics, MAR 9, 2022, View Source [SID1234609794]).
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NTLA-5001 is an autologous TCR-T cell therapy designed to target the Wilms’ Tumor (WT1) antigen, which is highly expressed in AML and many other hematologic and solid tumors. NTLA-5001 is currently being evaluated in a Phase 1/2a study in adults with persistent or recurrent AML who have previously received first-line therapy.
"The FDA’s decision to grant orphan drug designation for NTLA-5001 reflects the serious need for novel treatment options for people living with AML, a disease with notably poor long-term survival," said Intellia President and Chief Executive Officer John Leonard, M.D. "As part of our full-spectrum genome editing strategy, we seek to leverage our proprietary CRISPR/Cas9-based platform to engineer differentiated cell therapies targeting cancers for which there are currently limited or no treatment options. We look forward to advancing our investigational TCR-T cell therapy, NTLA-5001, through the clinic in hopes of improving future treatment options for patients in need."
The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user-fee exemptions and seven-year marketing exclusivity upon FDA approval.
About the NTLA-5001 Clinical Program
The Phase 1/2a study will evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy. The study includes a dose escalation and expansion phase, with up to 54 total participants. The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Visit clinicaltrials.gov (NCT05066165) for more details.
About NTLA-5001
NTLA-5001 is an investigational CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy in development for the treatment of all genetic subtypes of acute myeloid leukemia (AML). This autologous cell therapy candidate is designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is widely overexpressed in AML and other cancers. NTLA-5001 is Intellia’s first wholly owned ex vivo therapeutic candidate, developed using its proprietary cell engineering platform for the treatment of cancer. NTLA-5001 utilizes a WT1-targeting TCR identified in collaboration with IRCCS Ospedale San Raffaele. Based on preclinical results, Intellia believes its proprietary cell engineering platform will result in a pipeline of more efficacious and safer cell-based cancer therapies.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment. It is the most common type of acute leukemia in adults in the U.S., with more than 20,000 estimated new cases in 2021. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than 30%. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.