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Business Wire
03-06-2025
- Business
- Business Wire
ASCO Report of Pioneering Treatment of Lymphopenia with Significant Overall Survival Benefit in Advanced Pancreatic Cancer
CULVER CITY, Calif.--(BUSINESS WIRE)--ImmunityBio, Inc. (NASDAQ: IBRX) today announced results presented at ASCO 2025 of the first known treatment for lymphopenia with ANKTIVA and CAR-NK therapy. This data supports that reversal of lymphopenia, a well-established root cause of early mortality in patients with cancer across all tumor types, correlates with significant improved survival. While anemia and neutropenia have long been addressed by agents like Epogen and Neupogen, no therapy has ever existed for lymphopenia—until now. ANKTIVA (nogapendekin alfa inbakicept-pmln), an IL-15 superagonist approved in April 2024 for BCG-unresponsive non-muscle- invasive bladder cancer carcinoma in situ with or without papillary disease, represents the first lymphocyte-stimulating agent (LSA) capable of expanding lymphocytes critical for immunogenic cell death, such as natural killer (NK) and T cells. These findings emphasize the need for a therapy to overcome treatment induced lymphopenia with higher mortality as presented at ASCO 2025 by several institutions (Abstract # 8054, Satoskar et al. and Abstract # 2663, Saleh et al.) In a single-arm QUILT-88 clinical trial of 86 participants with third-to-sixth-line metastatic pancreatic cancer with very high tumor burden (CA19-9 levels exceeding 34,000 IU/ml), for which no therapy currently exists, patients received ANKTIVA subcutaneously in combination with off-the-shelf, ex-vivo infusion of CAR-NK cells (PD-L1 t-haNK) and low dose immuno-modulating chemotherapy. This first reported study of treating lymphopenia demonstrated significant differences in median overall survival in subjects whose lymphopenia was reversed (Absolute Lymphocyte Count: ALC ≥ 1,000). In 67 out of 86 subjects, reversal of lymphopenia was achieved and median overall survival was significantly prolonged compared to those who remained in severe lymphopenia with P-value 0.005, HR: 0.46 (0.26, 0.80) in Figure 1. In subjects with lymphopenia rescue and a lower tumor burden (less than the median CA19-9 of 34,000 IU/mL), the median overall survival in these very advanced metastatic pancreatic cancer patients exceeded 10 months (Figure 2). These findings of improved survival in pancreatic cancer patients with lower tumor burden point to the potential of further prolonged overall survival in pancreatic cancer patients if treated in the first line or neoadjuvant stages of disease. Highlighting the importance of lymphopenia reversal, Oncologist published a peer-reviewed paper titled ' Recurrent pancreatic cancer treated with N-803 and PD-L1 t-haNK followed by an EGFR-targeted nanocell drug conjugate,' demonstrating that in a patient with 2nd line metastatic pancreatic cancer treated with the full Cancer BioShield platform—including ANKTIVA, CAR-NK cells (PD-L1 t-haNK), and antigen-targeting adenoviruses—remained in remission for over six years and maintains a high quality of life at the date of this release. The expanded access authorization announced yesterday enables patients across all solid tumor types who have exhausted first-line therapy including chemotherapy, radiation, or immunotherapy to receive Anktiva as a lymphocyte stimulating agent to protect the immune system from the lymphogenic adverse effects of current standards of care. The ASCO Annual Meeting 2025 materials from ImmunityBio can be found below: 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. Abstract Text: Poster PDF: About the Cancer BioShield™ Platform The Cancer BioShield platform is a first-in-class immunotherapy strategy designed to restore immune competence by reversing lymphopenia—the loss of functional immune cells caused by cancer itself and by conventional treatments such as chemotherapy, radiation and immunotherapy. At its core is ANKTIVA® (nogapendekin alfa inbakicept-pmln), an IL-15 agonist approved for BCG-unresponsive non-muscle-invasive bladder cancer CIS with or without papillary disease, activates and 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. The platform employs a multi-modal approach: 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. Together, these components offer a comprehensive, novel, immune-restoring therapeutic platform aimed at not only expanding effector immune cells but also overcoming tumor-mediated immune suppression to support long-term disease control. The platform's effectiveness can be tracked through universally utilized, simple complete blood count (CBC): increases in absolute lymphocyte count (ALC) reflect ANKTIVA's lymphocyte-stimulating activity, while reductions in the neutrophil-to-lymphocyte ratio (NLR) demonstrate PD-L1 t-haNK's immunosuppressive neutrophil targeting. Low ALC and high NLR levels are laboratory measurements that have been extensively reported as predictive biomarkers of poor prognosis with early mortality across all tumor types 5,6. The data presented by ImmunityBio for the first time demonstrates that improving ALC and NLR correlates with significant enhanced overall survival and clinical benefit. About Lymphopenia and Absolute Lymphocyte Count (ALC) Lymphopenia—the loss of key immune cells such as NK, CD4+, and CD8+ T cells—is a common side effect of chemotherapy 1, radiation 2,3, and some immunotherapies 4. Unlike anemia and neutropenia, which have FDA-approved treatments like EPO and Neupogen, no therapy previously existed to treat this immune cell depletion. Lymphopenia weakens the immune system, increases infection risk, and is linked to early death across many cancer types 5. Low Absolute Lymphocyte Count (ALC) is a recognized poor prognostic marker. ANKTIVA ® is the first approved therapy to restore lymphocyte levels by activating and expanding NK and T cells—without increasing immunosuppressive T regulatory cells 7. More information on lymphopenia could be found on Twitter/X @DrPatSoonShiong articles here: References: Ray-Coquard I, et al. Lymphopenia as a prognostic factor for overall survival in advanced carcinomas, sarcomas, and lymphomas. Cancer Res. 2009 Jul 1;69(13):5383-91. doi: 10.1158/ Epub 2009 Jun 23. PMID: 19549917; PMCID: PMC2775079. Chen D, et al. Absolute Lymphocyte Count Predicts Abscopal Responses and Outcomes in Patients Receiving Combined Immunotherapy and Radiation Therapy: Analysis of 3 Phase 1/2 Trials. Int J Radiat Oncol Biol Phys. 2020 Sep 1;108(1):196-203. doi: 10.1016/ Epub 2020 Feb 7. Pike LRG, et al. The Impact of Radiation Therapy on Lymphocyte Count and Survival in Metastatic Cancer Patients Receiving PD-1 Immune Checkpoint Inhibitors. Int J Radiat Oncol Biol Phys. 2019 Jan 1;103(1):142-151. doi: 10.1016/ Epub 2018 Sep 15. PMID: 30227198. Lee, Y.J., et al. Peripheral lymphocyte count as a surrogate marker of immune checkpoint inhibitor therapy outcomes in patients with non-small-cell lung cancer. Sci Rep 12, 626 (2022). Ménétrier-Caux C., et al. Lymphopenia in Cancer Patients and its Effects on Response to Immunotherapy: an opportunity for combination with Cytokines? J Immunother Cancer. 2019 Mar 28;7(1):85. doi: 10.1186/s40425-019-0549-5. PMID: 30922400; PMCID: PMC6437964. Templeton AJ, et al. Prognostic role of neutrophil-to-lymphocyte (NLR) ratio in solid tumors: a systematic review and meta-analysis. J Natl Cancer Inst. 2014 May 29;106(6):dju124. doi: 10.1093/jnci/dju124. PMID: 24875653. FDA ANKTIVA Label, April 2024 - 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. 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 clinical trial data and potential results and implications to be drawn therefrom, the expectation that the EAP described herein will enable access to ANKTIVA for patients across all solid tumor types who have exhausted first-line therapy including chemo, radiation or immunotherapy, the RMAT designation as previously reported and potential results therefrom and regulatory submissions in connection therewith, the belief that ALC levels and NLR levels obtained from a CBC are predictors of clinical benefit and outcomes relating to overall survival, the belief that improving ALC levels and NLR levels correlates with enhanced overall survival and clinical benefit, the belief that reversal of lymphopenia correlates with improved survival, clinical trial and expanded access program enrollment, data and potential results to be drawn therefrom, anticipated components of ImmunityBio's Cancer BioShield platform, 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 alone or in combination with other therapeutic agents 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 and for potential applications beyond oncology, potential regulatory pathways and the regulatory review process and timing thereof, the application of the Company's science and platforms to treat cancers or develop cancer vaccines, immunotherapies and cell therapies that has the potential to change the paradigm in cancer care, 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 the FDA regulatory submission, filing and review process and the timing thereof, (ii) whether the RMAT designation will lead to an accelerated review or approval, of which there can be no assurance, (iii) risks and uncertainties regarding commercial launch execution, success and timing, (iv) risks and uncertainties regarding participation and enrollment and potential results from the expanded access clinical investigation program described herein, (v) whether clinical trials will result in registrational pathways and the risks, (vi) whether clinical trial data will be accepted by regulatory agencies, (vii) 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, (viii) potential delays in product availability and regulatory approvals, (ix) ImmunityBio's ability to retain and hire key personnel, (x) ImmunityBio's ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (xi) potential product shortages or manufacturing disruptions that may impact the availability and timing of product, (xii) ImmunityBio's ability to successfully commercialize its approved product and product candidates, (xiii) ImmunityBio's ability to scale its manufacturing and commercial supply operations for its approved product and future approved products, and (xiv) 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 the Company's Form 10-Q filed with the SEC on May 12, 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.
Yahoo
17-03-2025
- Business
- Yahoo
Kincell Bio Welcomes Lawrence (Larry) Pitcher as Chief Operating Officer to Lead the Preparation for Pivotal and Commercial Supply of Cell Therapies for Clients
RESEARCH TRIANGLE PARK, N.C., March 17, 2025--(BUSINESS WIRE)--Kincell Bio, a leader in cell therapy development and manufacturing, is excited to announce the appointment of Larry Pitcher as Chief Operating Officer (COO). Larry brings extensive experience in the advanced therapies Contract Development and Manufacturing Organization (CDMO) space, having had P&L responsibility, deep operational experience including facility expansions and key leadership roles. Most recently, Larry was VP and GM for Catalent's Gene Therapy Sites in MD, their largest operation, which accounted for a significant share of the company's annual revenue. While at Catalent, Larry scaled commercial manufacturing and operations, enhanced customer satisfaction, and drove double-digit growth in both revenue and profitability while expanding both clinical and commercial programs. Previously, Larry was at Thermo Fisher where he established a growing presence in advanced therapies across a network of sites including a new mRNA business in Italy, a cell therapy business and clinical and commercial microbial and plasmid DNA services in the U.S. Earlier in his career, Larry was GM and site head for Brammer Bio's viral vector business, general manager at RTI Surgical's German and French operations, and held various operational roles in Florida. Larry earned a B.S. in Industrial Engineering from Western Michigan University and an MBA from the University of Florida. "Larry joins the senior leadership team at Kincell Bio at a critical moment when client programs are moving into pivotal clinical studies and we are preparing for their near-term product launches at our RTP, NC site as well as expanding our process and analytical development and early clinical supply from our Gainesville, FL site," said Mark R. Bamforth, CEO of Kincell Bio. "I am delighted to join Kincell, which has a clear focus on solving technical challenges for their cell therapy clients, ensuring reliable supply, and ultimately supporting patients in need through operational excellence," said Larry Pitcher, COO at Kincell Bio. Kincell Bio is committed to advancing the field of cell therapy with tailored solutions that empower innovators to bring their therapies to market efficiently and effectively. About Kincell Bio Kincell Bio engineers cells into therapies. With manufacturing facilities located in Research Triangle Park, NC and Gainesville, FL, Kincell Bio is a contract development and manufacturing organization (CDMO) with the mission to streamline CMC development, apply expertise in analytical and process development and GMP manufacturing, testing and release from early clinical to pivotal studies and product launch. Kincell Bio is focused on supporting innovative companies developing immune cell therapies, including autologous and allogeneic CAR-T, CAR-NK and CAR-M programs, as well as developing expertise in stem cell products and iPSCs. For more information, visit View source version on Contacts For further information, please contact info@ Sign in to access your portfolio
Yahoo
05-03-2025
- Business
- Yahoo
ImmunityBio Receives FDA RMAT Designation for ANKTIVA® and CAR-NK for the Reversal of Lymphopenia in Patients Receiving Standard-of-Care Chemotherapy/Radiotherapy and in Treatment of Multiply Relapsed Locally Advanced or Metastatic Pancreatic Cancer
Regenerative Medicine Advanced Therapy (RMAT) designation follows clinical data of Absolute Lymphocyte Count (ALC) and significant Overall Survival (OS) correlations in QUILT trials across multiple tumor types, including third line or greater metastatic pancreatic cancer, checkpoint relapsed NSCLC, and supportive data from healthy volunteers The reversal of lymphopenia by ImmunityBio's IL-15 superagonist is consistent with the mechanism of action of ANKTIVA demonstrating proliferation and activation of NK cells, CD4+ T cells, CD8+ T cells and memory T cells without upregulation of suppressive T regulatory cells and approved in the ANKTIVA label Company intends to submit Biologic License Application (BLA) for the indication of reversal of lymphopenia in patients receiving standard-of-care chemotherapy and/or radiation and for the treatment of locally advanced or metastatic pancreatic cancer, which includes the first-in-class CAR-NK (PD-L1 t-haNK) ImmunityBio to provide data from fully enrolled clinical trials in metastatic pancreatic cancer (QUILT-88) and in checkpoint relapsed NSCLC (QUILT-3.055, NSCLC Cohort) patients, as well as lymphopenia reversal across multiple tumor types (QUILT-3.055, All Cohorts), with supportive data of lymphocyte proliferation in healthy volunteers (QUILT-1.004) In addition, ImmunityBio intends to file an Expanded Access Policy (EAP) for ANKTIVA and PD-L1 t-haNK in combination with standard of care chemotherapy/radiotherapy within 15 days and submit the protocol to the Agency CULVER CITY, Calif., February 28, 2025--(BUSINESS WIRE)--ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, today announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for ANKTIVA® and CAR-NK (PD-L1 t-haNK) for the reversal of Lymphopenia in Patients Receiving Standard-of-Care Chemotherapy/Radiotherapy and in Multiply Relapsed Locally Advanced or Metastatic Pancreatic Cancer. The complete blood count (CBC) is a standard assay widely used by oncologist to assess the status of the immune system following chemotherapy and radiation. To date, information of the cellular elements in the CBC assay provide information to the physician for the treatment of anemia, neutropenia and reduced platelet counts associated with the adverse events of chemotherapy and radiotherapy. Anemia, neutropenia and reduced platelet counts can be treated with currently approved therapies, including EPOGEN, NEUPOGEN and platelet transfusion, respectively. However, chemotherapy and radiation has also caused a reduction in the very cells necessary to kill cancer cells. This reduction in the lymphocytes by our standard of care also inhibits the induction of T cell memory in the absence of CD4+, CD8+ T cells. A treatment for the reversal of these adverse events of lymphopenia, induced by current standard-of-care, has eluded the industry for the past 50 years. ImmunityBio and its Founder Dr. Patrick Soon-Shiong developed a vision over the past decades that activation and proliferation of these key lymphocytes was necessary if we were to win the war against cancer and indeed even prevent cancer in subjects at high-risk such as with lynch syndrome with a cancer vaccine. The founder's vision reflecting the pursuit of addressing lymphopenia over the past decades will be updated in March. "RMAT designation for ANKTIVA combined with NK cells was applied for by the Founder in the initial 2017 IND. With the clinical results of the QUILT trials across multiple tumor types from 2017 to 2024, validating the hypothesis that high-dose chemotherapy and radiation induces lymphopenia and can be reversed by ANKTIVA together with off-the-shelf CAR-NK cells (PD-L1 t-haNK) resulting in prolongation of overall survival (OS), and enabling ImmunityBio to reapply for RMAT in 2025,"1 said Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific & Medical Officer of ImmunityBio. "I am so grateful for the FDA to have recognized the evolution of science and the need for adoption of 21st century medicine and cell therapy, particularly the role of NK cell therapy in our war against cancer as a universal therapy in cancer, and in the potential treatment of infectious diseases such as HIV, HPV and COVID. Today's designation of ANKTIVA and the first CAR-NK (PD-L1 T-haNK), both first-in-class molecules to activate lymphocytes within the body (via subcutaneous injection of ANKTIVA) and via ex-vivo infusion of off-the-shelf PD-L1 NK cells, is an inflection point and a paradigm change of how we could treat patients with cancer and viral infections. The absolute lymphocyte count (ALC) which has been largely ignored by physicians, since no therapy existed to address lymphopenia, could now be both a prognostic biomarker but more importantly, the potential as a therapeutic biomarker." "Multiple publications in the last five years have shown that patients with low lymphocyte counts, especially those with severe lymphopenia, have a statistically lower survival rate regardless of the tumor types.2-5 With this RMAT designation and the attributes of a RMAT designation including all Breakthrough Therapy Designation features and statutory ways to support Accelerated Approval, we will move rapidly to file the BLA for these authorized indications provided by the RMAT designation," said Soon-Shiong. "In addition, per the requirement under section 561A(f)(2) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), ImmunityBio will make publicly available the Expanded Access Policy of ANKTIVA and PD-L1 t-haNK in combination with standard-of-care chemotherapy/radiotherapy within 15 days." In the authorization letter, the FDA has committed to work closely with ImmunityBio to provide guidance and advice on generating the evidence needed to "support approval" of the indication above "in an efficient manner." 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 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 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit (Founder's Vision) and About CAR-NK (PD-L1 t-haNK) PD-L1 t-haNK is a human, allogeneic, stable clonal NK cell line generated from the parental aNK cell line (NK-92), manufactured by the Sponsor under cGMP conditions. Based on the demonstrated therapeutic efficacy of CAR targeting and on the important role of FcγR-mediated ADCC in the effectiveness of therapeutic IgG1 monoclonal antibodies, we hypothesized that modification of the parental aNK cell line to stably express both a PD-L1–targeted CAR and the high-affinity variant of CD16 would result in potent and selective antitumor activity. Myeloid-derived suppressor cells (MDSCs) express PD-L1 in concert with MHC-I loss to induce immune escape of tumors resistant or relapsed from chemo-immunotherapy including checkpoint inhibitors. Thus, there is a rationale for the combination of Anktiva (converting a cold tumor to hot tumor and rescuing T cells by re-expressing MHC-I) and PD-L1 t-haNK to overcome the immunosuppressive effect of TGFβ secreted by MDSCs. The ability to target both the tumor and MDSCs with off-the-shelf, outpatient based safe infusion of allogeneic CAR-NKs targeting PD-L1, was the basis for the development of this CAR-NK. Preclinical studies published in the J Immunotherapy Cancer 2020 "PD-L1 targeting high-affinity NK (t-haNK) cells induce direct antitumor effects and target suppressive MDSC populations" (Fabian et al. 2020) demonstrate that PD-L1 targeting of high affinity NK cells (PD-L1 t-haNK) induced direct anti-tumor effects in TNBC tumor cell lines and target suppressive MDSCs populations. About ImmunityBio ImmunityBio is a leading biotechnology company addressing cancer and infectious diseases by bolstering the natural immune system. The company is developing therapeutics that coordinate innate and adaptive immune responses, stimulating robust, multifunctional immunity and sustained immune memory that results in safe, long-term protection against disease recurrence. The company's lead asset is ANKTIVA® (nogapendekin alfa inbakicept-pmln) solution for intravesical use, approved in the U.S. by the Food and Drug Administration (FDA) indicated with Bacillus Calmette-Guérin (BCG) for the treatment of adult patients with BCG-unresponsive bladder carcinoma in situ (CIS) with or without papillary tumors. ANKTIVA's ability to activate natural killer (NK) cells, killer T cells, and memory T cells makes it a candidate 'backbone' therapy that may enhance the effectiveness of other therapies when used in combination in various tumor types. In a Phase 2 study, combining ANKTIVA with checkpoint inhibitor (CPI) therapy has shown potential to improve overall survival of patients with advanced or metastatic non-small cell lung cancer who have become resistant to CPI treatment. Further, Phase 1 findings in healthy participants have demonstrated ANKTIVA's potential to reverse lymphopenia, a severe depletion of key immune white blood cells, that is a frequent consequence of conventional radiation or chemotherapy in cancer patients. Based on the approved mechanism of action of proliferating and activating NK cells, CD4+, CD8+ T cells and memory T cells without proliferation of immuno-suppressive Treg cells, ANKTIVA has the potential to overcome the adverse events of chemotherapy and radiotherapy of lymphopenia. The company has received Regenerative Medicine Advanced Therapy (RMAT) designation for the development of ANKTIVA plus CAR-NK (PD-L1 t-haNK) for the reversal of lymphopenia for patients receiving standard-of-care chemotherapy/radiotherapy and in multiply relapsed locally advanced or metastatic pancreatic cancer. ANKTIVA is just one product of ImmunityBio's vertically-integrated R&D engine comprising state-of-the-art laboratories and production facilities supporting a range of next-generation immunotherapies (Immunotherapy 2.0, beyond checkpoint inhibitors), including cell therapies, and vaccine platforms, all of which are designed to be effective, safe, easy to administer, and to reduce or eliminate the need for invasive and burdensome treatments - such as high-dose chemotherapy - that are currently standard of care. For more information, visit (Founder's Vision) and connect with us on X (Twitter), Facebook, LinkedIn, and Instagram. About Regenerative Medicine Advanced Therapy (RMAT) The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including cell therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product's biologics license application (BLA). References: Fabian KP, Padget MR, Donahue RN, Solocinski K, Robbins Y, Allen CT, Lee JH, Rabizadeh S, Soon-Shiong P, Schlom J, Hodge JW. PD-L1 targeting high-affinity NK (t-haNK) cells induce direct antitumor effects and target suppressive MDSC populations. J Immunother Cancer. 2020 May;8(1):e000450. Rubinstein MP, Williams C, Mart C, Beall J, MacPherson L, Azar J, Swiderska-Syn M, Manca P, Gibney BC, Robinson MD, Krieg C, Hill EG, Taha SA, Rock AD, Lee JH, Soon-Shiong P, Wrangle J. Phase I Trial Characterizing the Pharmacokinetic Profile of N-803, a Chimeric IL-15 Superagonist, in Healthy Volunteers. J Immunol. 2022 Mar 15;208(6):1362-1370. doi: 10.4049/jimmunol.2100066. Epub 2022 Feb 28. PMID: 35228263. Guo Z, Zhang Z, Prajapati M, Li Y. Lymphopenia Caused by Virus Infections and the Mechanisms Beyond. Viruses. 2021 Sep 20;13(9):1876. Ménétrier-Caux C, Ray-Coquard I, Blay JY, Caux C. Lymphopenia in Cancer Patients and its Effects on Response to Immunotherapy: an opportunity for combination with Cytokines? J Immunother Cancer. 2019 Mar 28;7(1):85. Valero C, Lee M, Hoen D, Weiss K, Kelly DW, Adusumilli PS, Paik PK, Plitas G, Ladanyi M, Postow MA, Ariyan CE, Shoushtari AN, Balachandran VP, Hakimi AA, Crago AM, Long Roche KC, Smith JJ, Ganly I, Wong RJ, Patel SG, Shah JP, Lee NY, Riaz N, Wang J, Zehir A, Berger MF, Chan TA, Seshan VE, Morris LGT. Pretreatment neutrophil-to-lymphocyte ratio and mutational burden as biomarkers of tumor response to immune checkpoint inhibitors. Nat Commun. 2021 Feb 1;12(1):729. 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 RMAT designation referenced herein and potential results therefrom, the related anticipated EAP submission and timing thereof, the related anticipated BLA submission and timing thereof, 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/or PD-L1 t-haNK 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) whether the RMAT designation will lead to an accelerated review or approval, of which there can be no assurance, (ii) ImmunityBio's ability to submit the regulatory submissions referenced herein on the anticipated timeline or at all, (iii) additional risks and uncertainties related to the regulatory submission, filing and review process and the timing thereof, (iv) the ability of ImmunityBio to fund its ongoing and anticipated clinical trials, (v) whether clinical trials will result in registrational pathways, (vi) whether clinical trial data will be accepted by regulatory agencies, (vii) 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, (viii) potential delays in product availability and regulatory approvals, (ix) the risks and uncertainties associated with third party collaborations and agreements, (x) ImmunityBio's ability to retain and hire key personnel, (xi) ImmunityBio's ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (xii) potential product shortages or manufacturing disruptions that may impact the availability and timing of product, (xiii) ImmunityBio's ability to successfully commercialize its approved product and product candidates, (xiv) ImmunityBio's ability to scale its manufacturing and commercial supply operations for its approved product and future approved products, and (xv) 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 19, 2024 and the Company's Form 10-Q filed with the SEC on November 12, 2024, 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 ImmunityBio Contacts: Investors Hemanth Ramaprakash, PhD, MBA ImmunityBio, Inc. +1 Media Sarah Singleton ImmunityBio, Inc. +1 Sign in to access your portfolio
Los Angeles Times
05-03-2025
- Health
- Los Angeles Times
ImmunityBio Receives FDA RMAT Designation for ANKTIVA and CAR-NK (PD-L1 t-haNK) to Achieve Reversal of Lymphopenia in Pancreatic Cancer Patients
Culver City-based ImmunityBio, Inc., a leading immunotherapy company, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for the company's ANKTIVA and CAR-NK (PD-L1 t-haNK) for the reversal of Lymphopenia in patients receiving standard-of-care chemotherapy/radiotherapy and in multiply relapsed locally advanced or metastatic pancreatic cancer. The complete blood count (CBC) is a standard assay widely used by oncologists to assess the status of the immune system following chemotherapy and radiation. To date, information on the cellular elements in the CBC assay provides information to the physician for the treatment of anemia, neutropenia and reduced platelet counts associated with the adverse events of chemotherapy and radiotherapy. Anemia, neutropenia and reduced platelet counts can be treated with currently approved therapies, including EPOGEN, NEUPOGEN and platelet transfusion, respectively. However, chemotherapy and radiation have also caused a reduction in the very cells necessary to kill cancer cells. This reduction in the lymphocytes by the standard of care also inhibits the induction of T cell memory in the absence of specific (CD4+, CD8+) T cells. A treatment for the reversal of these adverse events of lymphopenia, induced by current standard of care, has eluded the industry for the past 50 years. ImmunityBio and its founder Dr. Patrick Soon-Shiong developed a vision over the past decades that activation and proliferation of these key lymphocytes was necessary to win the war against cancer and indeed even prevent cancer in subjects at high risk. 'With the clinical results of the QUILT trials across multiple tumor types from 2017 to 2024, validating the hypothesis that high-dose chemotherapy and radiation induces lymphopenia and can be reversed by ANKTIVA together with off-the-shelf CAR-NK cells (PD-L1 t-haNK) resulting in prolongation of overall survival, and enabling ImmunityBio to reapply for RMAT in 2025,' said Dr. Soon-Shiong. 'I am so grateful for the FDA to have recognized the evolution of science and the need for adoption of 21st-century medicine and cell therapy, particularly the role of NK cell therapy in our war against cancer as a universal therapy in cancer, and in the potential treatment of infectious diseases such as HIV, HPV and COVID. Today's designation of ANKTIVA and the first CAR-NK (PD-L1 T-haNK), both first-in-class molecules to activate lymphocytes within the body (via subcutaneous injection of ANKTIVA) and via ex-vivo infusion of off-the-shelf PD-L1 NK cells, is an inflection point and a paradigm change of how we could treat patients with cancer and viral infections. The absolute lymphocyte count (ALC) which has been largely ignored by physicians, since no therapy existed to address lymphopenia, could now be both a prognostic biomarker and more importantly, the potential as a therapeutic biomarker.' 'Multiple publications in the last five years have shown that patients with low lymphocyte counts, especially those with severe lymphopenia, have a statistically lower survival rate regardless of the tumor types,' added Dr. Soon-Shiong. 'With this RMAT designation and the attributes of an RMAT designation including all Breakthrough Therapy Designation features and statutory ways to support Accelerated Approval, we will move rapidly to file the BLA for these authorized indications provided by the RMAT designation. In addition, per the requirement under the Federal Food, Drug, and Cosmetic Act, ImmunityBio will make publicly available the Expanded Access Policy of ANKTIVA and PD-L1 t-haNK in combination with standard-of-care chemotherapy/radiotherapy within 15 days.' In the authorization letter, the FDA has committed to work closely with ImmunityBio to provide guidance and advice on generating the evidence needed to 'support approval' of the indication 'in an efficient manner.'
Associated Press
21-02-2025
- Business
- Associated Press
CRBU DEADLINE MONDAY: ROSEN, NATIONAL TRIAL LAWYERS, Encourages Caribou Biosciences, Inc. Investors to Secure Counsel Before Important February 24 Deadline in Securities Class Action
New York, New York--(Newsfile Corp. - February 21, 2025) - WHY: Rosen Law Firm, a global investor rights law firm, reminds purchasers of securities of Caribou Biosciences, Inc. (NASDAQ: CRBU) between July 14, 2023 and July 16, 2024, both dates inclusive (the 'Class Period'), of the important February 24, 2025 lead plaintiff deadline. SO WHAT: If you purchased Caribou securities during the Class Period you may be entitled to compensation without payment of any out of pocket fees or costs through a contingency fee arrangement. WHAT TO DO NEXT: To join the Caribou class action, go to or call Phillip Kim, Esq. toll-free at 866-767-3653 or email [email protected] for information on the class action. A class action lawsuit has already been filed. If you wish to serve as lead plaintiff, you must move the Court no later than February 24, 2025. A lead plaintiff is a representative party acting on behalf of other class members in directing the litigation. WHY ROSEN LAW: We encourage investors to select qualified counsel with a track record of success in leadership roles. Often, firms issuing notices do not have comparable experience, resources, or any meaningful peer recognition. Many of these firms do not actually litigate securities class actions, but are merely middlemen that refer clients or partner with law firms that actually litigate the cases. Be wise in selecting counsel. The Rosen Law Firm represents investors throughout the globe, concentrating its practice in securities class actions and shareholder derivative litigation. Rosen Law Firm achieved the largest ever securities class action settlement against a Chinese Company at the time. Rosen Law Firm was Ranked No. 1 by ISS Securities Class Action Services for number of securities class action settlements in 2017. The firm has been ranked in the top 4 each year since 2013 and has recovered hundreds of millions of dollars for investors. In 2019 alone the firm secured over $438 million for investors. In 2020, founding partner Laurence Rosen was named by law360 as a Titan of Plaintiffs' Bar. Many of the firm's attorneys have been recognized by Lawdragon and Super Lawyers. DETAILS OF THE CASE: According to the lawsuit, throughout the Class Period, defendants made false and misleading statements and/or failed to disclose that: (1) defendants overstated CB-010's safety, efficacy, and durability relative to approved autologous CAR-T cell therapies in treating patients with r/r B-NHL and/or LBCL, as well as CB-010's overall clinical results and commercial prospects; (2) Caribou was at significant risk of having insufficient cash, liquidity, and/or other capital to fund its current business operations, including preclinical research activities associated with the allogeneic CAR-NK platform; (3) the foregoing was likely to have a significant negative impact on Caribou's business and operations; and (4) as a result, defendants' public statements were materially false and misleading at all relevant times. When the true details entered the market, the lawsuit claims that investors suffered damages. To join the Caribou class action, go to call Phillip Kim, Esq. toll-free at 866-767-3653, or email [email protected] for information on the class action. No Class Has Been Certified. Until a class is certified, you are not represented by counsel unless you retain one. You may select counsel of your choice. You may also remain an absent class member and do nothing at this point. An investor's ability to share in any potential future recovery is not dependent upon serving as lead plaintiff. Attorney Advertising. Prior results do not guarantee a similar outcome. ------------------------------- Contact Information: Laurence Rosen, Esq. Phillip Kim, Esq. The Rosen Law Firm, P.A. 275 Madison Avenue, 40th Floor New York, NY 10016 Tel: (212) 686-1060 Toll Free: (866) 767-3653
