ImmunityBio Receives FDA RMAT Designation for ANKTIVA and CAR-NK (PD-L1 t-haNK) to Achieve Reversal of Lymphopenia in Pancreatic Cancer Patients

Culver City-based ImmunityBio, Inc., a leading immunotherapy company, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for the company’s ANKTIVA and CAR-NK (PD-L1 t-haNK) for the reversal of Lymphopenia in patients receiving standard-of-care chemotherapy/radiotherapy and in multiply relapsed locally advanced or metastatic pancreatic cancer.

The complete blood count (CBC) is a standard assay widely used by oncologists to assess the status of the immune system following chemotherapy and radiation. To date, information on the cellular elements in the CBC assay provides information to the physician for the treatment of anemia, neutropenia and reduced platelet counts associated with the adverse events of chemotherapy and radiotherapy. Anemia, neutropenia and reduced platelet counts can be treated with currently approved therapies, including EPOGEN, NEUPOGEN and platelet transfusion, respectively. However, chemotherapy and radiation have also caused a reduction in the very cells necessary to kill cancer cells.

This reduction in the lymphocytes by the standard of care also inhibits the induction of T cell memory in the absence of specific (CD4+, CD8+) T cells. A treatment for the reversal of these adverse events of lymphopenia, induced by current standard of care, has eluded the industry for the past 50 years. ImmunityBio and its founder Dr. Patrick Soon-Shiong developed a vision over the past decades that activation and proliferation of these key lymphocytes was necessary to win the war against cancer and indeed even prevent cancer in subjects at high risk.

“With the clinical results of the QUILT trials across multiple tumor types from 2017 to 2024, validating the hypothesis that high-dose chemotherapy and radiation induces lymphopenia and can be reversed by ANKTIVA together with off-the-shelf CAR-NK cells (PD-L1 t-haNK) resulting in prolongation of overall survival, and enabling ImmunityBio to reapply for RMAT in 2025,” said Dr. Soon-Shiong. “I am so grateful for the FDA to have recognized the evolution of science and the need for adoption of 21st-century medicine and cell therapy, particularly the role of NK cell therapy in our war against cancer as a universal therapy in cancer, and in the potential treatment of infectious diseases such as HIV, HPV and COVID. Today’s designation of ANKTIVA and the first CAR-NK (PD-L1 T-haNK), both first-in-class molecules to activate lymphocytes within the body (via subcutaneous injection of ANKTIVA) and via ex-vivo infusion of off-the-shelf PD-L1 NK cells, is an inflection point and a paradigm change of how we could treat patients with cancer and viral infections. The absolute lymphocyte count (ALC) which has been largely ignored by physicians, since no therapy existed to address lymphopenia, could now be both a prognostic biomarker and more importantly, the potential as a therapeutic biomarker.”

“Multiple publications in the last five years have shown that patients with low lymphocyte counts, especially those with severe lymphopenia, have a statistically lower survival rate regardless of the tumor types,” added Dr. Soon-Shiong. “With this RMAT designation and the attributes of an RMAT designation including all Breakthrough Therapy Designation features and statutory ways to support Accelerated Approval, we will move rapidly to file the BLA for these authorized indications provided by the RMAT designation. In addition, per the requirement under the Federal Food, Drug, and Cosmetic Act, ImmunityBio will make publicly available the Expanded Access Policy of ANKTIVA and PD-L1 t-haNK in combination with standard-of-care chemotherapy/radiotherapy within 15 days.”

In the authorization letter, the FDA has committed to work closely with ImmunityBio to provide guidance and advice on generating the evidence needed to “support approval” of the indication “in an efficient manner.”