ImmunityBio Quality-of-Life Study in BCG-Unresponsive Bladder Cancer Trial Indicates Improved Physical Function in the 71% Complete Responders Suggesting a Favorable Risk-Benefit Ratio for N-803 Plus BCG
- Positive PROs from the QUILT 3.032 trial of N-803 plus BCG for non-muscle invasive bladder cancer.
- Stability of physical function and global health in patients who completed questionnaires and reached month 24 on-study, supporting the safety and tolerability of the potential new treatment combination.
- FDA reviewing the Biologics License Application (BLA) for N-803 plus BCG, with a PDUFA date of April 23, 2024.
- None.
Insights
The QUILT 3.032 trial results are significant in the field of oncology, particularly for the treatment of non-muscle invasive bladder cancer (NMIBC). The study's focus on combining N-803, an interleukin-15 superagonist, with BCG (bacillus Calmette-Guerin) therapy is noteworthy because it represents a potential advancement in NMIBC treatment strategies. In clinical practice, the primary goal is to achieve a balance between efficacy and patient quality of life, which includes minimizing treatment-related side effects.
The reported patient outcomes indicating stability in physical function and global health over a two-year period are crucial. These outcomes suggest that the treatment may be as tolerable as BCG monotherapy, which has been the standard of care. It's important to note that the maintenance of physical function is a key factor in assessing the quality of life for cancer patients, as it allows them to continue with their daily activities and contributes to overall well-being.
Furthermore, the high response and cystectomy avoidance rates are particularly promising. Cystectomy, or the surgical removal of the bladder, is a significant procedure with a considerable impact on a patient's life. Therapies that can delay or avoid the need for such invasive surgery could be game-changers in NMIBC management.
The data from the QUILT 3.032 trial is quite compelling from a research perspective. The trial's design, focusing on a combination therapy approach for BCG-unresponsive NMIBC, addresses a substantial unmet need in this patient population. The reported 71% response rate and 89.2% cystectomy avoidance rate at the May 16, 2022 data cutoff are impressive and suggest that N-803 plus BCG could be a significant improvement over the current standard of care.
It's also worth discussing the implications of the trial's patient-reported outcomes (PROs). PROs are increasingly recognized as a vital component of clinical research because they provide direct insights into the patients' perspectives on their symptoms, functional status and overall quality of life. The stability of these PROs over 24 months suggests that the investigational therapy not only has potential efficacy but also maintains patients' quality of life during treatment.
The FDA's review of the Biologics License Application (BLA) for N-803 plus BCG is another critical aspect. The Prescription Drug User Fee Act (PDUFA) date set for April 23, 2024, will be a pivotal moment for the company, as approval could lead to a new standard of care for BCG-unresponsive NMIBC patients.
From a market perspective, the positive clinical trial results have significant implications for ImmunityBio, Inc. and the broader bladder cancer treatment market. The favorable safety profile and patient-reported outcomes could position N-803 plus BCG as a competitive therapy in a market where BCG-unresponsive patients have limited options. The potential FDA approval, indicated by the PDUFA date, could open a substantial market opportunity for ImmunityBio.
Bladder cancer is the 10th most common cancer and with a high percentage of newly diagnosed cases being NMIBC, the demand for effective treatments is considerable. The market dynamics are influenced by the need for therapies that not only improve survival rates but also maintain patients' quality of life. The trial results suggest that N-803 plus BCG could meet these needs, which may lead to increased adoption if approved.
Investor sentiment around ImmunityBio's stock (NASDAQ: IBRX) may be positively impacted by these developments, as the market often reacts to clinical trial outcomes and regulatory milestones. The company's valuation could see significant changes as the PDUFA date approaches and if the BLA is approved.
- Patient Reported Outcomes (PROs) based on a May 16, 2022 data cutoff in the QUILT 3.032 trial indicate stability of physical function and global health in patients who completed PRO questionnaires and reached month 24 on-study
- Overall, participants with complete responses (CRs) to combination therapy with the novel interleukin-15 (IL-15) superagonist N-803 and bacillus Calmette-Guerin (BCG) reported better physical function than those who did not achieve a CR at month 6
- Stability of these PROs supports the safety and tolerability of the potential new treatment combination
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Taken together with the positive durable
71% response and89.2% cystectomy avoidance rates in cohort A and a favorable safety profile at the May 16, 2022 data cutoff, along with the available PROs, support a favorable risk-benefit ratio for N-803 plus BCG in this patient population - The Food and Drug Administration (FDA) is currently reviewing the Biologics License Application (BLA) for N-803 plus BCG for the treatment of NMIBC CIS with a Prescription Drug User Fee Act (PDUFA) date of April 23, 2024
The published PROs based on a May 16, 2022 data cutoff indicate that both physical function and global health as self-reported by QUILT 3.032 participants with BCG-unresponsive NMIBC CIS (cohort A) or papillary disease (cohort B), remained stable over the 2-year course of the study for patients who completed the PRO questionnaires and reached 24 months on-study. In addition, overall both cohort A and B participants who reached month 24 on-study and also completed an NMIBC-specific questionnaire focusing on the challenges of bladder cancer, also reported no decline of their health or urinary tract-related symptoms while in the study. Overall, participants who achieved a complete response with the novel combination therapy reported better physical function by month six of the study than those who did not achieve a complete response.
“The self-reported stability of health and physical function over the course of the study by the participants reflect another aspect of safety and tolerability of this new combination therapy,” said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. “Taken together with the positive response rate in cohort A of over
The finding of relative stability of global health and physical function during the course of the study is similar to that reported by others for BCG monotherapy, suggesting the novel combination is as tolerable as treatment with BCG alone.
“Many current therapies for bladder cancer slow disease progression but can cause debilitating side effects,” said Principal Investigator Karim Chamie, M.D., Associate Professor of Urology at UCLA. “The data from the QUILT 3.032 Quality of Life study suggest that many patients not only have a durable response but also report no decline in physical function, which is very important for these patients.”
QUILT 3.032 Study Details
The ongoing phase 2/3 open-label multicenter registrational study QUILT 3.032 (NCT03022825) is evaluating the safety and efficacy of the investigational interleukin-15 superagonist N-803 (also known as Anktiva® and nogapendekin alfa inbakicept, NAI) in combination with a standard therapy for NMIBC, bacillus Calmette-Guerin (BCG), in patients who failed or in whom cancer returned after BCG monotherapy, and thus were diagnosed as BCG-unresponsive. The study comprises three cohorts, with cohort A enrolling patients with carcinoma in situ (CIS) with or without Ta/T1 disease and cohort B enrolling patients with high grade Ta/T1 papillary disease. Both cohorts A and B received combination N-803 plus BCG therapy. Cohort C patients, with CIS +/- Ta/T1 disease received N-803 monotherapy.
- The primary end point is the incidence of CR at the 3- or 6-month assessment visit for cohorts A and C, and the disease-free survival (DFS) rate at 12 months for cohort B.
- Durability, cystectomy avoidance, progression-free survival, disease-specific survival (DSS), and overall survival are secondary end points for cohort A.
- Cohort C was discontinued due to a low response rate with N-803 monotherapy, per study design.
- The FDA has accepted for review ImmunityBio’s resubmission of its biologics license application (BLA) for N-803 plus BCG for the treatment of BCG-unresponsive NMIBC CIS with or without Ta or T1 disease, and has set a user fee goal date (PDUFA date) of April 23, 2024.
Bladder cancer is the 10th most-commonly diagnosed cancer, with approximately
N-803 is investigational. Safety and efficacy have not been established by any Health Authority or Agency, including the FDA.
About ImmunityBio
ImmunityBio is a vertically-integrated, clinical-stage biotechnology company developing next-generation therapies and vaccines that bolster the natural immune system to defeat cancers and infectious diseases. The Company’s range of immunotherapy and cell therapy platforms, alone and together, act to drive and sustain an immune response with the goal of creating durable and safe protection against disease. ImmunityBio is applying its science and platforms to treating cancers, including the development of potential cancer vaccines, as well as developing immunotherapies and cell therapies that ImmunityBio believes sharply reduce or eliminates the need for standard high-dose chemotherapy. These platforms and their associated product candidates are designed to be more effective, accessible, and easily administered than current standards of care in oncology and infectious diseases. For more information, please visit Immunitybio.com.
Forward Looking Statements
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