ImmunityBio Files Supplemental BLA for NMIBC and Expands ANKTIVA Access for Lymphopenia Treatment
ImmunityBio, Inc. (NASDAQ: IBRX), a leading company specializing in immunotherapy, has recently made significant strides in advancing its therapeutic offerings. The company announced the completion of multiple key submissions to the U.S. Food and Drug Administration (FDA), including a Supplemental Biologics License Application (sBLA) for its immunotherapy, ANKTIVA® (nogapendekin alfa inbakicept-pmln), in combination with Bacillus Calmette-Guérin (BCG) therapy for the treatment of non-muscle invasive bladder cancer (NMIBC). In addition, ImmunityBio has submitted an Expanded Access Protocol (EAP) for ANKTIVA to address lymphopenia, a condition caused by cancer treatments that leaves patients vulnerable to infections and reduces their ability to fight cancer.
Supplemental Biologics License Application for BCG-Unresponsive NMIBC
In the first quarter of 2025, ImmunityBio completed the submission of a supplemental biologics license application (sBLA) to the FDA, proposing the use of ANKTIVA in combination with BCG therapy to treat BCG-unresponsive NMIBC, specifically targeting the papillary disease indication. This submission seeks to extend the clinical benefits of ANKTIVA beyond the current FDA-approved indication for carcinoma in situ (CIS), allowing more patients to benefit from a promising therapeutic option.
BCG therapy has long been the gold standard for treating NMIBC, particularly for high-risk patients. However, a significant portion of patients do not respond to BCG and require alternative treatment options. For these individuals, the potential of ANKTIVA in combination with BCG could provide an important opportunity to avoid the highly invasive procedure of radical cystectomy, which entails the surgical removal of the bladder. The morbidity and mortality associated with this procedure are substantial, making non-surgical alternatives a critical need.
ImmunityBio’s submission to the FDA includes compelling data demonstrating the efficacy of ANKTIVA in combination with BCG for patients with BCG-unresponsive papillary NMIBC. The data submitted suggests that a significant number of patients have achieved durable complete remissions, with results showing a probability of avoiding bladder removal of 88% for two years and 82% for three years. These promising results suggest that the combination of ANKTIVA and BCG could offer substantial clinical benefits, sparing patients from the challenges of a radical cystectomy and preserving their quality of life.
Pivotal Study Results and Disease-Free Survival Rates
A pivotal clinical trial published in NEJM Evidence further supports ImmunityBio’s application for ANKTIVA in combination with BCG. The study reported that the combination therapy led to a disease-free survival (DFS) rate of 55% at 12 months, 51% at 18 months, and 48% at 24 months in patients with papillary NMIBC. These rates underscore the potential for ANKTIVA to play a significant role in improving long-term outcomes for patients who are unresponsive to BCG monotherapy.
One of the most striking findings of the study was the high rate of cystectomy avoidance. With a median follow-up of 20.7 months, 93% of patients in the trial were able to avoid the need for bladder removal, a critical result for a patient population that would otherwise face high-risk surgery. The combination therapy is seen as a promising therapeutic option, especially considering that papillary disease is significantly more common than CIS—estimated to be 6-10 times more prevalent. This indicates that a large patient population could potentially benefit from ANKTIVA in combination with BCG.
In summary, ImmunityBio’s sBLA submission represents a major step forward for the treatment of BCG-unresponsive NMIBC. The data presented supports the therapeutic value of ANKTIVA as a critical addition to the treatment armamentarium for patients who would otherwise have limited options. If approved, this new indication could offer hope for many who are otherwise at risk of undergoing the traumatic and life-altering experience of radical cystectomy.
Expanded Access Protocol for Lymphopenia Treatment
In addition to its work in oncology, ImmunityBio has also made strides in addressing a significant unmet need in the treatment of lymphopenia. Lymphopenia refers to a condition characterized by a reduced number of natural killer (NK) cells and T cells, both of which are critical for immune function and fighting cancer. Lymphopenia is a common side effect of cancer treatments, including chemotherapy, radiation, steroids, and immune checkpoint inhibitors. Despite the critical nature of this condition, there are currently no approved therapies to counteract lymphopenia.
To address this gap, ImmunityBio has submitted an Expanded Access Protocol (EAP) to the FDA for ANKTIVA to treat lymphopenia. The company received a Regenerative Medicine Advanced Therapy (RMAT) designation for this indication, which is granted to promising regenerative medicines that aim to treat serious or life-threatening conditions. The EAP, if authorized, would enable patients and physicians to access ANKTIVA as part of their treatment regimen in combination with existing standards of care.
The submission is an important step toward improving the outcomes for cancer patients who experience lymphopenia as a result of their treatment. Without an effective remedy, these patients remain at risk for severe infections and other complications, which can jeopardize their health and overall survival. By offering ANKTIVA as a treatment option for lymphopenia, ImmunityBio aims to help restore critical immune function and improve the overall well-being of cancer patients undergoing aggressive therapies.
Positive Financial Momentum and Q1 2025 Sales Performance
ImmunityBio’s financial performance has seen significant positive momentum, driven by growing product sales and increasing demand for its immunotherapies. The company’s recently issued permanent J-code (J9028), which became effective in January 2025, has contributed to a notable increase in unit sales, with March 2025 sales volumes jumping 69% compared to February 2025. Furthermore, Q1 2025 unit sales have exceeded the total sales for all of fiscal year 2024, signaling a strong and continued upward trajectory for the company’s revenue generation.
In the first quarter of 2025, ImmunityBio reported an estimated $16.5 million in net product revenue, marking an impressive 129% increase over the $7.2 million in net revenue reported for the fourth quarter of 2024. These early estimates reflect the company’s growth, particularly in light of the J-code issuance and the increasing acceptance of its immunotherapy treatments in the market.
It is important to note that the figures disclosed in the press release are preliminary estimates, based on the information available as of the announcement date. The final figures for Q1 2025 will be disclosed in ImmunityBio’s upcoming Quarterly Report on Form 10-Q, which will include comprehensive financial data and any necessary adjustments to the preliminary numbers. Nonetheless, the reported growth indicates a strong and positive outlook for the company, with sustained sales growth expected as ImmunityBio continues to expand the reach of its therapies.
ImmunityBio is making significant progress in its mission to revolutionize the treatment landscape for cancer and immune-related conditions. The completion of its sBLA submission for ANKTIVA in combination with BCG for BCG-unresponsive NMIBC is a key milestone that could provide a breakthrough treatment option for a large patient population in need of alternatives to radical cystectomy. Additionally, the company’s submission of the EAP for ANKTIVA in lymphopenia represents an important opportunity to address a critical gap in cancer care, offering hope to patients who suffer from the debilitating effects of lymphopenia.
With positive momentum in both clinical development and product sales, ImmunityBio is well-positioned to continue its growth and contribute meaningfully to the advancement of immunotherapy. The company’s innovative approach and commitment to improving patient outcomes make it one to watch in the rapidly evolving field of immunotherapy and regenerative medicine.
As the FDA reviews ImmunityBio’s submissions and the company continues to build on its successes, the future looks bright for both the company and the patients it serves.
