In breaking news, clinical-stage biotechnology company ImmunityBio, Inc. (NASDAQ: IBRX) has announced acceptance by the FDA review of a Biologics License Application (BLA) for N-803 the company’s antibody cytokine fusion protein as a treatment for patients with BCG-unresponsive non-muscle-invasive bladder cancer carcinoma in situ (CIS) with or without Ta or T1 disease.
The acceptance is the first regulatory finding for N-803, which was granted Breakthrough Therapy and Fast Track designations as an IL-15 superagonist and submitted to the FDA for this usage in combination with Bacillus Calmette-Guérin (BCG) for this indication. The Prescription Drug User Fee Act (PDUFA) target date is May 23, 2023.
N-803 Anktiva & QUILT 3.032 Trial
The BLA submission is based on positive results from a series of studies of the investigational treatment, including the ongoing QUILT 3.032 trial. This trial showed positive results in which 71% of BCG-unresponsive NMIBC patients who had failed on previous therapies showed a complete response with a median duration of 26.6 months; cystectomy avoidance rate of 91% and 100% bladder cancer overall survival at 24 months with zero serious adverse events (SAE).
ImmunityBio’s first BLA to reach this stage of FDA acceptance for review was N-803 combined with BCG. This is a significant step toward ImmunityBio’s goal of replacing high-dose chemotherapy with immunotherapy in cancer treatment without requiring the use of toxic drugs.
N-803 plus BCG would be the first immunotherapy combination for this indication in 23 years that can be administered directly to the bladder (intravesically) to create natural killer cells and T cells if approved. It is a critical step in Patrick Soon-Shiong’s Nant Cancer Vaccine hypothesis, which was proposed by ImmunityBio.
The mechanism of action of N-803 is to boost NK and T cells, which are components of the adaptive and innate immune systems. N-803 provides a secondary boost to the immunological response induced by BCG for bladder cancer or by a checkpoint inhibitor for other indications via this mechanism.
In the QUILT 3.032 trial, 71% of patients who had previously failed on other therapies demonstrated an over 50% improvement in response and median duration compared to the FDA-approved alternatives Valrubicin and Pembrolizumab, a systemic checkpoint inhibitor therapy for this indication.
ImmunityBio is focused on developing next-generation medicines and vaccines that assist the body’s natural immune system in fighting cancer and infectious diseases. The company’s immunotherapy and cell therapy platforms, alone and together, aim to elicit an immune response that can last for years. These platforms and their associated product candidates are intended to be more efficient, accessible, and simple to use than existing oncology and infectious disease standards of care.
ImmunityBio’s clinical pipeline consists of 27 clinical trials—18 of which are in Phase 2 or 3 development—across 13 indications in liquid and solid tumors (including bladder, pancreatic, and lung cancers) and infectious diseases (including SARS-CoV-2 and HIV).
ImmunityBio’s lead cytokine fusion protein, N-803 (Anktiva™), is a novel interleukin-15 (IL-15) superagonist complex that has been granted Breakthrough Therapy and Fast Track Designations by the US Food and Drug Administration (FDA) for BCG-unresponsive CIS non-muscle invasive bladder cancer (NMIBC).
The firm has a GMP production capacity at size with cutting-edge cell manufacturing know-how and ready-to-scale facilities, as well as extensive and experienced R&D, clinical trial, and regulatory operations, and development teams.