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ImmunityBio, a prominent biotechnology company, has made significant strides in cancer and immune-related therapies with its groundbreaking development of ANKTIVA® and CAR-NK (PD-L1 t-haNK). On February 27, 2025, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation for these two promising treatments. This designation is pivotal for ImmunityBio, as it accelerates the development and approval process for ANKTIVA and CAR-NK in addressing lymphopenia caused by standard-of-care chemotherapy and radiotherapy, as well as in the treatment of multiply relapsed locally advanced or metastatic pancreatic cancer.
What is RMAT Designation?
The RMAT designation is granted by the FDA to cell and gene therapies that show significant potential in treating serious or life-threatening conditions. This designation is part of the FDA’s commitment to advancing innovative therapies that can address unmet medical needs. With the RMAT status, ImmunityBio’s ANKTIVA and CAR-NK treatments now benefit from features such as the potential for Accelerated Approval, regulatory support, and more efficient development pathways.
The Role of ANKTIVA® and CAR-NK in Reversing Lymphopenia
One of the primary challenges in cancer treatment, particularly after chemotherapy and radiotherapy, is lymphopenia — a dangerous reduction in immune cells, including critical lymphocytes, that hampers the body’s ability to fight cancer cells. Standard cancer treatments often induce lymphopenia, which weakens the immune system, making patients vulnerable to infections and limiting the effectiveness of ongoing cancer treatment.
ImmunityBio’s ANKTIVA®, a first-in-class interleukin-15 (IL-15) superagonist, has shown remarkable promise in reversing lymphopenia. ANKTIVA works by activating and proliferating natural killer (NK) cells, as well as CD4+ and CD8+ T cells — key immune cells involved in the destruction of cancer cells. In addition, ANKTIVA promotes the formation of memory T cells that retain long-lasting immune memory against tumor cells, enhancing the body’s immune response even after treatment.
The unique mechanism of ANKTIVA ensures that the body’s immune cells can multiply and respond effectively to cancer cells without promoting the activation of suppressive T regulatory cells, which are known to hinder immune responses. This ability to enhance immune function while preventing further immune suppression is central to its potential in reversing lymphopenia.
CAR-NK (PD-L1 t-haNK) for Pancreatic Cancer and Beyond
ImmunityBio has also developed CAR-NK (PD-L1 t-haNK), a novel, allogeneic NK cell therapy. PD-L1 t-haNK cells are engineered to target the PD-L1 protein on tumor cells, which is often exploited by tumors to evade the immune system. By targeting this protein, PD-L1 t-haNK cells are able to directly attack tumor cells and suppress the immune-evasive mechanisms tumors employ.
The combination of ANKTIVA and CAR-NK presents a potent approach to treating not only the side effects of cancer treatment, such as lymphopenia, but also stubborn cancers like pancreatic cancer. In particular, metastatic pancreatic cancer, which has a poor prognosis, is one of the most difficult cancers to treat. The use of ANKTIVA and CAR-NK in this context offers a new avenue for patients who have relapsed or are resistant to standard treatments.
ImmunityBio’s clinical trials, including the QUILT-88 study in metastatic pancreatic cancer, have demonstrated promising data, showing correlations between lymphocyte proliferation and improved overall survival (OS). This data further supports the potential of these therapies in both reversing lymphopenia and providing a more effective treatment for aggressive cancers.
A Vision for the Future of Cancer Therapy
Dr. Patrick Soon-Shiong, the founder of ImmunityBio, has long been a proponent of addressing the root causes of cancer’s ability to evade the immune system. His vision has been instrumental in the development of therapies like ANKTIVA and CAR-NK. Dr. Soon-Shiong’s decades of work have been focused on activating and proliferating key immune cells to reverse lymphopenia and create long-lasting immune responses that could prevent cancer recurrence.
This RMAT designation is a major milestone in this journey, affirming the validity of the company’s approach and its potential to revolutionize cancer treatment. ImmunityBio’s therapies aim to break new ground in the field of immunotherapy, especially by offering an innovative treatment strategy that goes beyond traditional chemotherapy, providing hope for patients with limited treatment options.
What’s Next for ImmunityBio?
With the RMAT designation secured, ImmunityBio plans to submit a Biologic License Application (BLA) for ANKTIVA and CAR-NK. The company is also poised to file an Expanded Access Policy (EAP) within the next 15 days, allowing more patients to access these therapies.
As ImmunityBio continues to advance the development of ANKTIVA and CAR-NK, the company is focused on improving the treatment landscape for cancers like pancreatic cancer and enhancing the immune system’s response to conventional therapies. If successful, these therapies could be transformative for cancer patients, offering new hope for survival and a better quality of life after treatment.
By addressing the devastating effects of lymphopenia and enhancing the immune response, these therapies have the potential to shift how we approach cancer treatment and immunotherapy for patients at great risk.
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