FDA authorizes Expanded Access Program of Anktiva for lymphopenia

The FDA has authorized an Expanded Access Program (NCT06956547) for use of nogapendekin alfa inbakicept-pmln (NAI; Anktiva) to manage lymphopenia in adult patients with refractory or relapsed solid tumors, including genitourinary tumors, who have progressed after first-line standard-of-care treatment with chemotherapy, radiation, and/or checkpoint inhibitors, ImmunityBio announced in a news release.¹

The chemo-immunotherapy regimen is being assessed in the the phase 2 QUILT-88 study.

The Expanded Access Program is specifically assessing the use of ImmunityBio’s Cancer BioShield platform, which includes NAI at its core. NAI was approved by the FDA in 2024 for the treatment of patients with BCG-unresponsive non–muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary disease.

As part of the Cancer BioShield program, NAI “proliferates natural killer (NK) cells and CD4+ and CD8+ T cells, restoring lymphocyte levels critical for immunosurveillance, immunogenic cell death, and long-term tumor control,” ImmunityBio explained.

According to the company, the platform includes the following:

• “In-vivo stimulation: Subcutaneous administration of ANKTIVA expands NK and T cells, boosting anti-tumor immunity.
• Ex-vivo targeted cytotoxicity: Off-the-shelf PD-L1 t-haNK CAR-NK cells are engineered to target and eliminate PD-L1–expressing tumor cells and immunosuppressive neutrophils (myeloid-derived suppressor cells), enhancing anti-tumor specificity and reducing immune evasion.
• Memory Cytokine-Enriched Natural Killer (M-ceNK) cell therapy: M-ceNK cells are developed via cytokine activation and expansion of autologous and allogeneic NK cells collected through apheresis, potentially providing long-term immune memory and sustained cytotoxic capacity.”

The BioShield platform was granted a Regenerative Medicine Advanced Therapy designation by the FDA in February 2025 for the management of lymphopenia.

For decades, epoetin alfa (epogen) and filgrastim (neupogen) have been used to treat patients with chemotherapy- and radiation-induced anemia and neutropenia, respectively. However, there has been no comparable option available for the management of lymphopenia.

In correspondence with Urology Times®, Soon-Shiong explained, “To this day, there's never been a molecule, a fusion protein, that rescues lymphocytes, the NK cells and T cells, which are the key cells for killing cancer and generating memory. For the first time, this is Anktiva.”

Data on the Cancer BioShield

Data from the phase 2 QUILT-88 study (NCT04390399) were recently presented at the 2025 American Society of Clinical Oncology Annual Meeting in Chicago, Illinois.² Overall, the trial was assessing the use of NAI and PD-L1-targeted high-affinity NK cell therapy (PD-L1 t-haNK) in combination with low-dose chemotherapy in patients with locally advanced or metastatic pancreatic cancer.

Data showed that the chemo-immunotherapy protocol led to a median overall survival (OS) of 6.2 months (95% CI, 5.0 to 7.1) in the 3^rd line setting and 5.7 months (95% CI, 4.3 to 6.4) in the 3^rd to 6^th line setting. According to the authors, a median OS exceeding 6 months is roughly 2 months greater than the OS achieved by other therapies in the 3^rd to 5^th line setting.

Additionally, a higher absolute lymphocyte count (ALC) was associated with greater survival. Median OS was 7.1 months in patients with an ALC of 1.045 x 109 cells/L or higher and a CA19-9 less than 4079.6 U/mL compared with a median OS of 3.1 months for patients with an ALC less than 1.045 x 109 cells/L and a CA19-9 of 4079.6 U/mL or higher (HR, 3.6; P < .001).

Grade 3 or higher treatment-emergent adverse events occurred in 95% of patients. These were largely attributed to chemotherapy, according to the authors.

“Lymphopenia has long been recognized as a major driver and predictor of early mortality in cancer—yet until now, it has remained unaddressed,” said Patrick Soon-Shiong, MD, founder, executive chairman, and global chief scientific and medical officer of ImmunityBio, in the news release.¹ “This FDA authorization allows all patients with solid tumors suffering from immune collapse following first-line therapy of chemo, radiation, or immunotherapy to access ANKTIVA. The survival benefit we observed at ASCO 2025 in 3rd to 6th line advanced metastatic pancreatic cancer confirms that restoring lymphocyte levels—rather than depleting them—can change the course of disease.”

REFERENCES

1. ImmunityBio receives FDA Expanded Access authorization for landmark treatment of lymphopenia with ANKTIVA, the Cancer BioShield Platform, in patients with solid tumors. News release. ImmunityBio. June 2, 2025. Accessed June 7, 2025. https://immunitybio.com/immunitybio-receives-fda-expanded-access-authorization-for-landmark-treatment-of-lymphopenia-with-anktiva-the-cancer-bioshield-platform-in-patients-with-solid-tumors/

2. Seery T, Nangia C, McKean H, et al. Association of lymphopenia rescue and CA19-9 levels with overall survival following IL-15 superagonist N-803 and PD-L1 t-haNK chemo-immunotherapy for 3rd line or greater metastatic pancreatic cancer. 2025. doi:10.1200/JCO.2025.43.16_suppl.2542. Abstract 2542