Latest news with #BCG-unresponsive
Business Wire
05-05-2025
- Business
- Business Wire
ImmunityBio Requests an Urgent Meeting With FDA to Address the Change in the Agency's Unambiguous Guidance on Jan 2025 to Submit a sBLA for NMIBC BCG Unresponsive Papillary Disease, Following an Inconsistent Refusal to File Letter on May 2, 2025
CULVER CITY, Calif.--(BUSINESS WIRE)--ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, today announced that the Company received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for the supplemental biologics license application (sBLA) for use of ANKTIVA plus Bacillus Calmette-Guerin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for the indication of papillary disease. This RTF letter was received despite reaching unanimous guidance and encouragement at the in-person January 2025 meeting from the leadership of the Agency, including from CBER, CDER and OCE to submit this sBLA. At this meeting all key decision makers were specifically asked and unanimously confirmed that ImmunityBio should submit the sBLA as soon as possible based on the data in the single-arm trial. Relying on this unanimous guidance the Company submitted the sBLA in March 2025. The Company has already requested an urgent meeting to resolve the inconsistencies between the directives provided at the January Meeting and receipt of the RTF letter. ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of BCG unresponsive NMIBC with Papillary tumors with CIS (Cohort A). In the same clinical trial (QUILT-3.032) long term results of patients with Papillary tumors without CIS (Cohort B), was submitted as a sBLA. The RTF letter referenced herein does not impact this prior approval of CIS +/- Papillary in BCG unresponsive patients. The Agency leaders present at the January meeting unambiguously invited the Company to expeditiously submit an sBLA for the NMIBC papillary indication based on the strength of the clinical response and long-term duration of follow-up data of the Papillary without CIS cohort of the QUILT 3.032 study. Data was also presented of the long term follow-up Phase 1 results which demonstrated Complete Response and Disease Free Survival in both CIS and Papillary disease, with patients in on-going cystectomy free state at 10 years. In addition disease-specific overall survival rate of 99% at 12 months and 96% at 36 months, in the BCG unresponsive patients with Papillary disease without CIS was discussed. To our knowledge, this is the most durable data to date in the Papillary setting for bladder preservation. In March 2025, the Company completed its submission to the FDA of the sBLA for the use of ANKTIVA plus BCG in BCG-unresponsive NMIBC in the papillary indication. On May 2, 2025, and notwithstanding the FDA's invitation to submit the sBLA articulated by its leadership at the January Meeting, the FDA delivered an RTF letter. The Company together with its consultants who attended the January 2025 meeting were shocked by this inconsistent response and have requested an urgent meeting with the Agency to resolve this issue. 'Our commitment to NMIBC patients in the papillary indication and our belief in ANKTIVA's potential based on the strength of the clinical response and long duration of 5-year follow-up remains unchanged, despite our receipt of a refusal to file letter regarding our supplemental BLA,' said Dr. Patrick Soon-Shiong, the Company's Founder, Executive Chairman and Global Chief Scientific and Medical Officer. 'We are fully determined to work with the FDA as quickly as possible, including having already requested a Type A meeting, to explore the best path forward. We would also welcome an FDA Advisory Committee meeting as part of the regulatory process going forward. We presented our data at the recent 2025 American Urological Association (AUA) meeting and ANKTIVA+BCG was considered best in class and best in disease by the thought leaders in attendance, when compared to all the therapies currently approved or in development. Patients with BCG Unresponsive Papillary disease, face a life changing and life-threatening prospect of a total radical cystectomy, as well as the danger of the disease progressing from non-muscle invasive to muscle invasion with consequent progression and mortality. With the data presented of 99% disease specific survival at 12 months and over 82% patients not requiring bladder removal, it is essential that these patients be provided this treatment option, especially with the safety profile of ANKTIVA+BCG and the already approved indication of Papillary disease with CIS. The Company presented these data to the Agency in person in January and the Agency leaders present unanimously encouraged the company to expeditiously submit a supplemental BLA for this indication.' Dr. Soon-Shiong further stated: 'The Agency must explain the confounding inconsistency of approving ANKTIVA+BCG for patients with Papillary with CIS disease, while refusing to file the sBLA for patients with Papillary without CIS disease—even though both groups were part of the same trial, in the same high-risk population of BCG-unresponsive non-muscle invasive bladder cancer. In both cases, patients received the same surgical procedure for the Papillary component, the same therapy at the same dose, with the same excellent tolerability and meaningful clinical benefit: over 82% bladder-sparing and over 96% disease-specific survival at 36 months, as shown in Figure 1. On behalf of patients facing a potential loss of a vital organ and high risk of progression of disease, I urge the Agency to reconsider and act now.' 'I actively participated in the January 2025 meeting at which the leadership of the Agency present at that meeting unanimously supported and encouraged ImmunityBio to submit results from the single arm trial, QUILT 3.032 as a supplemental BLA because of the high-risk of progression and metastasis these patients with BCG unresponsiveness face. The consensus was reached by all present, including me, because of the lack of satisfactory alternatives in this desperately ill population at high-risk of losing their bladder – a life threatening and life changing procedure. Thus, I was startled to learn the ImmunityBio had received a 'Refuse to File' letter which is in my opinion rife with regulatory inaccuracies. I recommend that the company seek a rapid meeting with the Agency to resolve this issue and minimize the delay and threat to well-being of patients who could benefit significantly from avoiding a cystectomy,' said Dr. Rachel Sherman, former FDA Principal Deputy Commissioner, who pioneered single arm trials at the height of the HIV epidemic, and was responsible for developing the expedited pathways at the Agency to address life threatening and serious diseases on behalf of the American public during her 30 year career at the agency. 'Furthermore, it is incomprehensible to me that the FDA refuses to file a supplemental BLA, stating the study is not sufficient to support a regulatory review, when it has already approved a product based on that very same study in essentially the same indication and population. As stated above, these patients are suffering from a disease with a high risk of morbidity and mortality in the very short term – no delay should be tolerated,' added Dr. Sherman. About the QUILT-3.032 Study QUILT-3.032 (NCT03022825) is a Phase II/III, open-label, single-arm, multicenter study of intravesical BCG plus ANKTIVA or ANKTIVA only in patients with BCG unresponsive high grade non-muscle invasive bladder cancer (NMIBC). All participants receive BCG plus ANKTIVA (Cohorts A and B) or ANKTIVA only (Cohort C) via a urinary catheter in the bladder for 6 consecutive weeks (initial induction treatment period). After the first disease assessment, eligible patients receive either a 3-week maintenance course or a 6-week re-induction course (second treatment period) at Month 3. Eligible patients will continue to receive maintenance treatment in the third treatment period at Months 6, 9, 12, and 18. Eligible patients have the option to receive maintenance treatment in the fourth treatment period at Months 24, 30, and 36. The study duration is 60 months. Cohort A (N=100) includes patients with histologically confirmed BCG-unresponsive NMIBC CIS with or without papillary disease. The primary endpoint is biopsy-confirmed complete response (CR) rate at any time. Secondary endpoints include duration of CR, progression-free survival (PFS), time to cystectomy, safety and overall survival. FDA Approved this indication in 2024 in which eligibility included patients with Papillary disease. Cohort B (N=80) enrolled participants with histologically confirmed BCG-unresponsive high-grade Papillary disease without CIS. The primary endpoint is a response of a disease-free rate at 12 months. Secondary endpoints include disease-free survival DFS and disease-specific survival, PFS, time to cystectomy, safety and overall survival. About ANKTIVA® The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response. ANKTIVA is a first-in-class IL-15 receptor agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo. ANKTIVA was approved by the FDA in 2024 with BCG for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer with CIS with or without papillary tumors. For more information, visit (Founder's Vision) and About ImmunityBio ImmunityBio is a vertically integrated 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. Designated an FDA Breakthrough Therapy, ANKTIVA is the first FDA-approved immunotherapy for non-muscle invasive bladder cancer CIS that activates natural killer cells, T cells, and memory T cells for a long-duration response. The Company 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 we believe sharply reduce or eliminate 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, visit (Founder's Vision) and connect with us on X (Twitter), Facebook, LinkedIn, and Instagram. References: Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, such as statements regarding discussions and meetings with the U.S. FDA, the company's submission of the sBLA for use of ANKTIVA plus BCG in BCG-unresponsive NMIBC for the indication of papillary disease and potential results therefrom, the FDA's delivery of a refusal to file letter regarding the aforementioned sBLA submission, potential next steps, decisions and timeline related and requirements thereof, potential Type A meeting with the FDA regarding the sBLA and timing thereof and results therefrom, potential Advisory Committee meeting with the FDA regarding the sBLA and timing thereof and results therefrom, clinical trial data and potential results to be drawn therefrom, the development of therapeutics for cancer and infectious diseases, potential benefits to patients, potential treatment outcomes for patients, the described mechanism of action and results and contributions therefrom, potential future uses and applications of ANKTIVA and use in cancer vaccines and across multiple tumor types, and ImmunityBio's approved product and investigational agents as compared to existing treatment options, among others. Statements in this press release that are not statements of historical fact are considered forward-looking statements, which are usually identified by the use of words such as 'anticipates,' 'believes,' 'continues,' 'goal,' 'could,' 'estimates,' 'scheduled,' 'expects,' 'intends,' 'may,' 'plans,' 'potential,' 'predicts,' 'indicate,' 'projects,' 'is,' 'seeks,' 'should,' 'will,' 'strategy,' and variations of such words or similar expressions. Statements of past performance, efforts, or results of our preclinical and clinical trials, about which inferences or assumptions may be made, can also be forward-looking statements and are not indicative of future performance or results. Forward-looking statements are neither forecasts, promises nor guarantees, and are based on the current beliefs of ImmunityBio's management as well as assumptions made by and information currently available to ImmunityBio. Such information may be limited or incomplete, and ImmunityBio's statements should not be read to indicate that it has conducted a thorough inquiry into, or review of, all potentially available relevant information. Such statements reflect the current views of ImmunityBio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about ImmunityBio, including, without limitation, (i) risks and uncertainties regarding commercial launch execution, success and timing, (ii) risks and uncertainties regarding market access initiatives and timing, (iii) whether the FDA will accept the Company's request for a Type A meeting and/or Advisory Committee Meeting, (iv) whether the FDA will permit the resubmission of the sBLA and the requirements thereof, (v) uncertainties regarding the timeline of the FDA's review of these submissions even if accepted for review and filing, (vi) whether the FDA will ultimately approve the sBLA, or other submissions in a timely matter, or at all, of which there can be no assurance, (vii) risks and uncertainties regarding limited resources at the FDA and potential delays associated therewith, (viii) whether clinical trials will result in registrational pathways and the risks and uncertainties regarding the regulatory submission, filing, review and approval process, (ix) whether clinical trial data will be accepted by regulatory agencies, (x) the ability of ImmunityBio to continue its planned preclinical and clinical development of its development programs through itself and/or its investigators, and the timing and success of any such continued preclinical and clinical development, patient enrollment and planned regulatory submissions, (xi) potential delays in product availability and regulatory approvals, (xii) ImmunityBio's ability to retain and hire key personnel, (xiii) ImmunityBio's ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (xiv) potential product shortages or manufacturing disruptions that may impact the availability and timing of product, (xv) ImmunityBio's ability to successfully commercialize its approved product and product candidates, (xvi) ImmunityBio's ability to scale its manufacturing and commercial supply operations for its approved product and future approved products, and (xvii) ImmunityBio's ability to obtain, maintain, protect, and enforce patent protection and other proprietary rights for its product candidates and technologies. More details about these and other risks that may impact ImmunityBio's business are described under the heading 'Risk Factors' in the Company's Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 3, 2025, and in subsequent filings made by ImmunityBio with the SEC, which are available on the SEC's website at ImmunityBio cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof. ImmunityBio does not undertake any duty to update any forward-looking statement or other information in this press release, except to the extent required by law.
Yahoo
29-04-2025
- Health
- Yahoo
New data from J&J's bladder cancer drug-device trial strengthens support for NDA
Johnson & Johnson (J&J) has reported more data supporting the efficacy of its intravesical drug release system TAR-200 in patients with Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk non-muscle-invasive bladder cancer (HR-NMIBC). Data, announced during the Paradigm-Shifting, Practice-Changing Clinical Trials in Urology plenary session at the American Urological Association (AUA) Annual Meeting 2025, showed a high response rate in one of the cohorts from the Phase IIb SunRISe-1 trial (NCT04640623). J&J announced new data from both cohort two and cohort four at the conference. TAR-200, trademarked as GemRIS, is a drug-device combination product that continuously delivers gemcitabine chemotherapy directly to the bladder over a week. It can be inserted without the need for anaesthesia or sedation. J&J acquired TAR-200 and the drug delivery technology during its purchase of Taris Biomedical in 2019. In the new data from cohort two, which enrolled 85 BCG-unresponsive NMIBC patients with carcinoma in situ, 82.4% achieved a complete response (CR), with this translating into sustained disease control, with 52.9% of responders maintaining CR at one year. In the same cohort, the median duration of response (DOR) was 25.8 months and at 12 months, 86.6% of patients who responded to treatment remained cystectomy-free. Meanwhile, patients in cohort four, 52 HR-NMIBC patients with papillary disease-only, 85.3% and 81.1% disease-free survival (DFS) rates at six and nine months. Also, 94.2% of patients avoided cystectomy at a median follow-up of 12.8 months. J&J also reported progression-free (PFS) and overall survival (OS) rates of 95.6% and 98% at nine months, respectively. In both cohorts, TAR-200, the treatment was well-tolerated, with most adverse events (AEs) being mild urinary symptoms. These findings indicate that TAR-200 offers a highly effective and durable treatment option for patients with certain types of BCG-unresponsive HR-NMIBC. Most treatment-related AEs were mild and manageable. Between both cohorts, seven patients discontinued treatment due to treatment-related AEs, and there were no treatment-related deaths. Disease area leader of bladder cancer for J&J Innovative Medicine, Dr. Christopher Cutie, said: 'Bladder cancer is one of the ten most common cancers worldwide, yet treatment options have remained largely unchanged for over 40 years, leaving patients with few choices if initial BCG therapy does not work. TAR-200 is designed to allow for sustained delivery of medication directly into the bladder through a brief and routine procedure, which benefits patients. These data now show patients can remain cancer-free for a meaningful period of time, marking a significant step forward for those facing this challenging disease.' The new data, announced on Saturday 26 April comes after J&J filed a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for TAR-200 in January 2025 based on previous data announced from the SunRISe-1 trial. The application is for the use of TAR-200 in NMIBC patients with carcinoma in situ who are BCG-unresponsive, with or without papillary tumours. This submission is being reviewed through the Real-Time Oncology Review (RTOR) programme, which allows the FDA to review data before the complete application is formally submitted. Earlier results from cohort two were presented at the European Society of Medical Oncology (ESMO) Congress 2024 and the AUA 2024 Meeting. TAR-200 did not, however, show benefit in a Phase III trial in patients with muscle-invasive bladder cancer (MIBC) not undergoing radical cystectomy. The Phase III SunRISe-2 study (NCT04658862) was terminated by J&J after an independent data monitoring committee said the trial was unlikely to meet its primary endpoint of bladder intact-event free survival (BI-EFS). The Phase II SunRISe-4 trial, also in patients with MIBC, is ongoing. The SunRISe-1 trial is ongoing, and TAR-200 is also being investigated in NMIBC in two Phase III studies, SunRISe-3 (NCT05714202) and SunRISe-5 (NCT06211764). Also at the AUA Annual Meeting 2025, Pfizer presented positive data from a Phase III trial of sasanlimab and BCG, with the combination therapy improving event-free survival (EFS) in BCG-naïve high-risk NMIBC patients compared to subjects who were only given BCG, meeting the trial's primary endpoint. "New data from J&J's bladder cancer drug-device trial strengthens support for NDA" was originally created and published by Medical Device Network, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Business Insider
28-04-2025
- Business
- Business Insider
Johnson & Johnson announces new data from Cohort 2 of Phase 2b SunRISe-1
Johnson & Johnson announced new data from Cohort 2 of the pivotal Phase 2b SunRISe-1 study evaluating TAR-200-an intravesical gemcitabine releasing system-for patients with certain types of bladder cancer. The findings demonstrate the highest complete response rate without reinduction with more than half of responders remaining cancer-free for at least 12 months. These results highlight the potential of TAR-200 as a breakthrough for people with Bacillus Calmette-Guerin-unresponsive, high-risk non-muscle-invasive bladder cancer, HR-NMIBC, with carcinoma in situ, CIS, with or without papillary tumors who are ineligible or refuse radical cystectomy. As of March 2025, 82.4 percent of the 85 enrolled patients in the study achieved a complete response, meaning their cancer was undetectable following treatment. This high response rate translated into sustained disease control, with 52.9 percent of responders maintaining complete response at one year. The median duration of response was 25.8 months, indicating that many patients remained cancer-free for over two years without the need for reinduction therapy. At 12 months, 86.6 percent of responders remained cystectomy-free. Importantly, the treatment was well-tolerated, with most adverse events being mild urinary symptoms. These findings show that TAR-200 offers a highly effective and durable treatment option for patients with certain types of BCG-unresponsive HR-NMIBC. Stay Ahead of the Market:
Business Wire
24-04-2025
- Business
- Business Wire
U.S. FDA Approves Second Drug Product Manufacturing Facility for ADSTILADRIN ® (nadofaragene firadenovec-vncg)
PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals announced today the U.S. Food and Drug Administration (FDA) approved a state-of-the-art drug product manufacturing hub in Parsippany, NJ, for its intravesical non-replicating gene therapy ADSTILADRIN ® (nadofaragene firadenovec-vncg). The approval expands Ferring's manufacturing capabilities to three cutting-edge sites dedicated to bringing ADSTILADRIN to patients and secures a final $200 million payment from Royalty Pharma (Nasdaq: RPRX) as part of a royalty-based financing agreement announced in 2023. ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the FDA for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). Bladder cancer is the sixth most diagnosed cancer in the United States, and the majority of patients are diagnosed with non-muscle invasive disease. 1 'The FDA approval of our new manufacturing facility for ADSTILADRIN represents our unwavering dedication to delivering high-quality, innovative therapies at scale,' said Armin Metzger, Executive Vice President, Chief Technical Operations Officer, Ferring Pharmaceuticals. 'This expansion and diversification of our manufacturing footprint will further ensure stable and sustainable supply of ADSTILADRIN to meet the anticipated growth in global demand.' Located on Ferring's U.S. campus in Parsippany, NJ, the new state-of-the-art manufacturing site features a cutting-edge manufacturing suite, fully integrated with specialized modern technology and equipment to produce another source of supply for ADSTILADRIN. The 12,000 square foot facility features renewable energy solutions such as waste heat recovery with heat pumps and solar energy, complementing Ferring's commitment to protect the environment by reducing its impact on the planet. ' ADSTILADRIN has transformed the treatment landscape for BCG-unresponsive bladder cancer patients and drives Ferring's continued growth in uro-oncology, ' said Bipin Dalmia, Global Head of Uro-Oncology and Urology Franchise, Ferring Pharmaceuticals. 'The growing body of clinical evidence for ADSTILADRIN — including the recently announced independent real-world data 2 and data from our Japan Phase 3 trial 3 — underscores the impact of this therapy. The FDA's approval of this additional manufacturing site is a testament to our commitment to make ADSTILADRIN globally available to every bladder cancer patient who needs it.' About ADSTILADRIN ADSTILADRIN ® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder's cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body's own natural defenses against the cancer. ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment. 4 The pivotal Phase 3 study met its primary endpoint, with more than half (51%) of the 98 patients (95% CI [41%, 61%]) with CIS with or without papillary tumors (±Ta/T1) achieving a complete response (CR) by three months (full inclusion criteria published on NCT02773849). 4 Earlier this month, Ferring announced results from an ongoing Phase 3 trial of ADSTILADRIN in BCG-unresponsive CIS ± high-grade Ta/T1 patients (n=20) in Japan. At 3 months, 75% (15/20) of patients achieved a complete response. 2 Overall, 80% of participants experienced a drug–related AE, all of which were either Grade 1 (84.2%) or Grade 2 (15.8%). The data from Japan add to the growing body of evidence, including recent real-world data presented by the Mayo Clinic that showed a 79% CR rate, demonstrating consistent effectiveness and safety when patients are treated with ADSTILADRIN at three months (19/24 patients with CIS +/- Ta/T1). 3 Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days. 5 About Ferring Uro-Oncology Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X. About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall. 6 In the United States, bladder cancer is the sixth most common cancer, 1 fourth among men, 7 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025. 1 Historically, 75% of bladder cancer presents as NMIBC. 8 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease. 9 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder). 10 INDICATION ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product. WARNINGS AND PRECAUTIONS: Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy. Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals. DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration. USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose. ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination). You are encouraged to report negative side effects of prescription drugs to FDA. Visit or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING. Please click to see the full Prescribing Information. About Ferring Pharmaceuticals Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees. For more information, please visit call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X. References: American Cancer Society. Cancer Facts & Figures 2025. Available at: Accessed April 22, 2025. Moyer J, Durant A, Nguyen M. Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Presented at the Annual Meeting of the American Society of Clinical Oncology GU, February 2025. Inoue K, Kikuchi E, Nishiyama H, Nasu Y, et al. Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non–Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial. Presented at the 112 th Annual Meeting of the Japanese Urological Association, April 22, 2025. Available at: ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: Accessed April 22, 2025. Ferring Access Support benefits investigations for commercial and government-insured patients through March 15, 2024. Urology Care Foundation. Non-muscle Invasive Bladder Cancer. Available at: Accessed April 22, 2025. National Cancer Institute. Cancer Statistics. Available at: Accessed April 22, 2025. Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94. Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. doi: 10.3390/curroncol31020079. National Comprehensive Cancer Network. Bladder Cancer (Version 1.2025). Available at: Accessed April 22, 2025. More information is available at the following:
Yahoo
21-04-2025
- Business
- Yahoo
ImmunityBio Announces FDA Submissions of Supplemental BLA for NMIBC Papillary Disease and for Expanded Access of ANKTIVA® to Treat Lymphopenia
Company to Provide Regulatory, Sales, and Platform Updates at Investor Day including: In Q1, the company submitted a supplemental Biologics License Application (sBLA) for use of ANKTIVA® plus Bacillus Calmette-Guérin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) for the indication of papillary disease. In Q2, the company submitted to the U.S. Food and Drug Administration (FDA) an Expanded Access Protocol (EAP) for ANKTIVA for the treatment of lymphopenia as a BioShield against the adverse effects of chemotherapy, radiation and checkpoint inhibitors, following authorization of a Regenerative Medicine Advanced Therapy (RMAT) designation for this indication in Q1. With a permanent J-code (J9028) awarded in January 2025, ImmunityBio's Q1 2025 ANKTIVA unit sales volume grew 150% over unit sales volume in Q4 2024. For the three-month period ended March 31, 2025, ImmunityBio achieved estimated net product revenue of approximately $16.5 million, surpassing net product revenue of $7.2 million in the prior quarter, a 129% quarter-over-quarter increase. ANKTIVA sales momentum continues to trend upward in 2025, with sales volume in March representing a 69% increase month-over-month from February. Fireside chats with thought leaders covering ImmunityBio's platform to be held during the investor day program. CULVER CITY, Calif., April 15, 2025--(BUSINESS WIRE)--ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, today announced that it has completed multiple submissions to the FDA including an sBLA for BCG-unresponsive NMIBC in papillary disease and an EAP for ANKTIVA® (nogapendekin alfa inbakicept-pmln) for the treatment of lymphopenia. Supplemental Biologics License Application (sBLA): In Q1, ImmunityBio completed the submission to the FDA of an sBLA for the use of ANKTIVA plus BCG in BCG-unresponsive NMIBC in the papillary indication. Subject to regulatory approvals, the addition of the papillary indication expands the patient population benefiting from this therapy beyond the currently approved indication of bladder carcinoma in situ (CIS) with or without papillary disease and allows more patients to avoid the high morbidity and mortality associated with radical cystectomy. The data submitted to the FDA included efficacy results demonstrating durable complete remissions in patients with BCG unresponsive NMIBC papillary disease. In 88% and 82% of subjects, the probability of avoiding surgical removal of the bladder was achieved for as long as 2 and 3 years respectively, following treatment with ANKTIVA plus BCG. The mortality and morbidity associated with a radical total cystectomy is high and this long-term bladder sparing therapy has the potential to provide a significant benefit and quality of life to patients suffering from BCG unresponsive papillary disease. In a pivotal study published in NEJM Evidence, BCG plus ANKTIVA resulted in a disease-free survival (DFS) rate of 55% at 12 months, 51% at 18 months, and 48% at 24 months in participants with papillary NMIBC. In addition, patients receiving the novel treatment achieved a 93% avoidance of cystectomy (surgical removal of the bladder) with a median follow up of 20.7 months. This combination immunotherapy wherein ANKTIVA rescues BCG efficacy, currently approved in the BCG-unresponsive carcinoma in situ (CIS) indication, may provide an effective therapeutic option for papillary patients who did not respond to BCG alone and face the prospect of a radical cystectomy. Papillary disease is estimated to be approximately 6-10 times more common than bladder cancer CIS, representing a large patient population that may benefit from ANKTIVA plus BCG. Expanded Access Protocol for ANKTIVA in the Treatment of Lymphopenia: The company also announced it has submitted to the FDA an EAP to make available ANKTIVA for the treatment of lymphopenia. Lymphopenia is the loss of natural killer cells and T cells, the very cells necessary to fight cancer. To date, no treatment exists to overcome lymphopenia which is induced by the cancer itself and by the standards of care including chemotherapy, radiation, steroids and checkpoint inhibitors. ImmunityBio received designation from the Agency for Regenerative Medicine Advanced Therapy (RMAT) for the indication of ANKTIVA to treat lymphopenia. The EAP, subject to authorization, would provide early access to patients and physicians desiring ANKTIVA in combination with standards of care. Update of Product Revenue, Net Preliminary Results of Operations: With the issuance of the permanent J-code (J9028) in January 2025, ImmunityBio has seen increased sales momentum supporting a trend of increases month-over-month as well as quarter-over-quarter, with March unit sales volume increasing 69% over February, and Q1 2025 unit sales exceeding unit sales achieved for all of FY 2024. ImmunityBio earned net product revenue of approximately $16.5 million during the three-month period ended March 31, 2025, which represented an increase of 129% over the $7.2 million of net revenue earned during the fourth quarter of 2024. The amounts reported in this press release reflect the company's preliminary estimates based solely upon information available to it as of the date of this press release, and the amounts reported are not a comprehensive statement of its financial results or position as of March 31, 2025. Any actual amounts that the company reports in its Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, will be subject to its financial closing procedures and any final adjustments that may be made prior to the time its financial results for the period ended March 31, 2025 are finalized. As a result, these preliminary estimates may differ materially from the actual results that will be reflected in the company's consolidated financial statements for the quarter when they are completed and publicly disclosed. Update on ImmunityBio Platforms Fireside chats with Dr. Patrick Soon-Shiong and the following Key Opinion Leaders (KOLs) discussing the science and current status of ImmunityBio platforms are expected to take place during the Investor Day program: Dr. Christopher Pieczonka – Chief Executive Officer, Associated Medical Professionals of New York & Corporate Director of Clinical Research of US Urology Partners Dr. Steven Finkelstein – National Director of Radiation Oncology, US Urology Partners. Director of the Center of Advanced Radiation Excellence (CARE) and Director Radiation Oncology Research Dr. Mark Lanasa – Senior Vice President, Chief Medical Officer, Solid Tumors, BeiGene Dr. Jennifer Buell – President & Chief Executive Officer, MiNK Therapeutics Dr. Krishnansu Tewari – Gynecologic Oncology, Obstetrics & Gynecology at UC Irvine Health Dr. David Kerr - Professor of Cancer Medicine Genetics and Genomics, University of Oxford Dr. Timothy Henrich – Professor, School of Medicine UC San Francisco Dr. Carlos Cordon-Cardo – Chairman for the Mount Sinai Health System Dept. of Pathology The live stream can be found at: Participant Listening (Listen Only) 1-844-539-3703 or 1-412-652-1273 About ImmunityBio ImmunityBio is a vertically-integrated 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. Designated an FDA Breakthrough Therapy, ANKTIVA is the first FDA-approved immunotherapy for non-muscle invasive bladder cancer CIS that activates natural killer cells, T cells, and memory T cells for a long-duration response. The Company 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 we believe sharply reduce or eliminate 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. 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Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, such as statements regarding the company's estimated product revenue for Q1 2025 and certain monthly and quarterly sales unit volume and other information regarding operating results and prospects, commercialization activities, momentum and market data, the company's submission of the sBLA for use of ANKTIVA plus BCG in BCG-unresponsive NMIBC for the indication of papillary disease and potential results therefrom as well as regulatory review process, decisions and timeline related thereto, the company's submission of the EAP to provide ANKTIVA for preventing or reversing lymphopenia and potential results therefrom as well as regulatory review process, decisions and timeline related thereto, clinical trial and expanded access program enrollment, data and potential results to be drawn therefrom, the development of therapeutics for cancer and infectious diseases, potential treatment outcomes for patients, the described mechanism of action and results and contributions therefrom, potential future uses and applications of ANKTIVA for the prevention or reversal of lymphopenia, potential future uses and applications of ANKTIVA alone or in combination with other therapeutic agents across multiple tumor types and indications, potential regulatory pathways and the regulatory review process and timing thereof, potential expansion of patient populations or benefits to patients, potential market sizes, and the application of the Company's science and platforms to treat cancers or develop cancer vaccines, immunotherapies and cell therapies that reduce or eliminate the need for standard high-dose chemotherapy. Statements in this press release that are not statements of historical fact are considered forward-looking statements, which are usually identified by the use of words such as "anticipates," "believes," "continues," "goal," "could," "estimates," "scheduled," "expects," "intends," "may," "plans," "potential," "predicts," "indicate," "projects," "seeks," "should," "will," "strategy," and variations of such words or similar expressions. Statements of past performance, efforts, or results of our preclinical and clinical trials, about which inferences or assumptions may be made, can also be forward-looking statements and are not indicative of future performance or results. Forward-looking statements are neither forecasts, promises nor guarantees, and are based on the current beliefs of ImmunityBio's management as well as assumptions made by and information currently available to ImmunityBio. Such information may be limited or incomplete, and ImmunityBio's statements should not be read to indicate that it has conducted a thorough inquiry into, or review of, all potentially available relevant information. Such statements reflect the current views of ImmunityBio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about ImmunityBio, including, without limitation, (i) whether the FDA will accept the sBLA and other regulatory submissions referenced herein for review and filing, (ii) uncertainties regarding the timeline of the FDA's review of these submissions even if accepted for review and filing, (iii) whether the FDA will ultimately approve the sBLA, expanded access protocol or other submissions in a timely matter, or at all, of which there can be no assurance, (iv) risks and uncertainties regarding limited resources at the FDA and potential delays associated therewith, (v) whether the clinical trials and/or expanded access programs will result in registrational pathways and the risks and uncertainties regarding the regulatory submission, review and approval process, (vi) whether clinical trial data will be accepted by regulatory agencies in support of the submissions referenced herein or otherwise, (vii) the company's financial closing procedures and whether the estimated results reported herein will differ from the results ultimately reported by the company in its quarterly report on Form 10-Q for Q1 2025, (viii) whether the RMAT designation received and previously reported by the company will lead to an accelerated review or approval, of which there can be no assurance, (ix) risks and uncertainties regarding commercial launch execution, success and timing, and market access initiatives, (x) the ability of ImmunityBio to fund its ongoing and anticipated clinical trials, (xi) the ability of ImmunityBio to continue its planned preclinical and clinical development of its development programs through itself and/or its investigators, and the timing and success of any such continued preclinical and clinical development, patient enrollment and planned regulatory submissions, (xii) potential delays in product availability, regulatory approvals, and reimbursement decisions, (xiii) ImmunityBio's ability to retain and hire key personnel, (xiv) ImmunityBio's ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (xv) potential product shortages or manufacturing disruptions that may impact the availability and timing of product, (xvi) ImmunityBio's ability to successfully commercialize its approved product and product candidates, (xvii) ImmunityBio's ability to scale its manufacturing and commercial supply operations for its approved product and future approved products, and (xviii) ImmunityBio's ability to obtain, maintain, protect, and enforce patent protection and other proprietary rights for its product candidates and technologies. More details about these and other risks that may impact ImmunityBio's business are described under the heading "Risk Factors" in the Company's Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 3, 2025, and in subsequent filings made by ImmunityBio with the SEC, which are available on the SEC's website at ImmunityBio cautions you not to place undue reliance on any forward looking statements, which speak only as of the date hereof. ImmunityBio does not undertake any duty to update any forward-looking statement or other information in this press release, except to the extent required by law. View source version on Contacts Investors Hemanth Ramaprakash, PhD, MBA+ Media Sarah Singleton+
