CAMBRIDGE, Mass. – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, today announced that the U.S. Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application for NTLA-5001, the company’s first wholly-owned ex vivoCRISPR genome editing candidate for the treatment of cancer. NTLA-5001 is an autologous T cell receptor (TCR)-T cell therapy engineered to target the Wilms’ Tumor (WT1) antigen for the treatment of all genetic subtypes of acute myeloid leukemia (AML). Intellia intends to initiate patient screening by year-end in a Phase 1/2a study evaluating NTLA-5001 in adults with persistent or recurrent AML who have previously received first-line therapy.
“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. NTLA-5001 is our first wholly-owned ex vivo candidate to enter the clinic, and we expect to initiate this first-in-human study in adults with AML by year-end. 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,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Our study is an important first step toward improving treatment for people living with this aggressive form of cancer. AML is the most common type of acute leukemia in adults, that, despite currently available treatments, has a five-year survival rate of less than 30 percent.”
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 will contain a dose escalation and expansion phase, with up to 54 participants. The dose-escalation phase of the study will include two independent arms of up to three cohorts: Arm 1 will consist of adults with AML with lower disease burden, defined as those with less than 5% AML blasts in bone marrow, while Arm 2 will consist of adults with AML with higher disease burden, defined as those greater than or equal to 5% AML blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. More information about the study will be available at clinicaltrials.gov.
In addition to the U.S., Intellia has also submitted a regulatory application to the U.K. for NTLA-5001.
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, with more than 20,000 estimated new cases in 2020. Despite currently available treatments for AML, the five-year overall survival rate for patients remains less than thirty percent. AML, along with other cancer types, is often characterized by overexpression of the Wilms’ Tumor 1 (WT1) antigen.
NTLA-5001 is a 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 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. 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 Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics using CRISPR/Cas9 technology. To fully realize the transformative potential of CRISPR/Cas9, Intellia is pursuing two primary approaches. The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues. Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of CRISPR/Cas9 to create new classes of genetic medicine. Learn more at intelliatx.com. Follow us on Twitter @intelliatweets.
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