Memory Cytokine-Enhanced Natural Killer Cells Show Promising Results in Leukemia Patients; Data Support ImmunityBio’s Scaled m-ceNK Clinical Program

On March 2, 2022 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported study results that further demonstrate the potential for use of memory-like cytokine-enriched natural killer (m-ceNK) cells for the treatment of cancer (Press release, ImmunityBio, MAR 2, 2022, View Source [SID1234609366]). ImmunityBio has successfully scaled the process of generating over 20 billion m-ceNK cells from a single extraction of white blood cells from a donor or patient, and reports promising clinical data supporting the ongoing development of memory NK cells for the treatment of both liquid and solid tumors of all types.

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In a Phase 2 study sponsored by Washington University School of Medicine (NCT02782546), white blood cells retrieved from HCT donors to treat acute myeloid leukemia (AML) patients, were stimulated with cytokines (IL-12, -15, and -18) to form memory-like NK cells, and were re-infused to the patients, who also received ImmunityBio’s IL-15 superagonist Anktiva (N-803). The cytokine-stimulated NK cells safely augmented a leading treatment for AML-—HLA-haploidentical hematopoietic cell transplantation. The data showed that the cytokine-stimulated NK cells were persistent, showed increased production of interferon-gamma and functionality, and led to a complete response in 87% of patients after Day 28. The results were recently published in a peer-reviewed article ("Hematopoietic cell transplantation donor-derived memory-like NK cells functionally persist after transfer into patients with leukemia") in Science Translational Medicine.

NK cells are innate immune cells that are under investigation as a cell therapy for multiple types of cancer. A challenge of NK cell-based therapies, however, is ensuring that the cells persist and remain functional long-term. This study showed that memory-like NK cells derived from the same donor as the hematopoietic cell transplantation (HCT) persisted for at least two months after transplantation and were highly functional ex vivo. Together, these findings support the use of memory NK cells as a component of HCT for leukemia.

"This important study adds to the growing body of data that shows the cancer-fighting ability of NK cells can be enhanced by stimulating expansion and persistence of these cells," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "The study also showed that stimulated memory NK cells are effective with only a single infusion when given along with Anktiva support. To facilitate this approach, ImmunityBio has successfully scaled cytokine enrichment and expansion of m-ceNK cells, so cancer patients can receive multiple infusions of these potent and persistent m-ceNK cells generated from a single donation of white blood cells."

About Memory Cytokine-Enhanced NK Cells (m-ceNK):
ImmunityBio has successfully enriched and expanded NK cells obtained from peripheral blood of donors using a technique called apheresis to generate NK cells with a memory-like phenotype, which exhibit both high cytotoxicity and increased interferon-gamma production. These m-ceNK cells can be generated from an individual donor for autologous cell therapy, but have also been generated as an allogeneic product from cord blood. In addition to the high potential for enhanced efficacy, m-ceNK cells can be infused easily in an outpatient setting.

ImmunityBio has developed a novel method of m-ceNK cells production that yields multiple clinical-dose forms from a single apheresis (white blood cell collection) using the company’s proprietary NANT 001 Bioreactor (GMP-in-a-Box), thereby alleviating pressures on supply of starting material. An optimized cryopreservation protocol for maximum shelf-life and potency upon recovery was also established, a necessity for any off-the-shelf product. ImmunityBio is leveraging their ability to generate m-ceNK cells with potent cytotoxicity, increased IFN-gamma production, proliferative capacity, activation surface markers and memory response to establish a propriety method for generation, expansion, and cryopreservation of these cells for autologous use.

The QUILT 3.076 Study of Cryopreserved M-ceNK cells
Cryopreserved m-ceNK cells in combination with Anktiva (N-803) will be tested in a 2-part Phase 1 study (NCT04898543) designed to evaluate safety in subjects with locally advanced or metastatic solid tumors; solid tumors comprise approximately 90% of adult cancers and 40% of all cancers in children, according to the American Cancer Society. The study will compare the quantity and characteristics of m-ceNK cells collected and cytokine-enriched from newly diagnosed patients who have not received prior treatment to m-ceNK cells generated from patients who have received at least two prior treatments for their cancer.

The study consists of two cohorts and there will be 10 participants in each cohort. Cohort 1 includes participants with newly-diagnosed, high-risk solid tumors who have not received prior treatment; and cohort 2 includes participants with relapsed/refractory (r/r) solid tumors who have progressive disease after receiving ≥ 2 prior therapies. Participants will be enrolled in the two cohorts simultaneously.

Participants in Cohort 1 will participate in apheresis collection of lymphocytes (part A) and will not receive any investigational therapy in this study.
Participants in Cohort 2 will undergo an apheresis collection of lymphocytes (part A) prior to receiving 4 infusions of M-ceNK on days 1, 8, 15 and 22 along with N-803 on days 1 and 15.

Propanc Biopharma’s Cancer Stem Cell Technology Offers Renewed Hope to Achieve a Total Victory Against Metastatic Cancer

On March 2, 2022 Propanc Biopharma, Inc. (OTCQB: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that among the large and rapidly growing stem cell therapeutics sector, the Company’s cancer stem cell technology, PRP, offers renewed hope to achieve a total victory in the fight against cancer (Press release, Propanc, MAR 2, 2022, View Source [SID1234609365]). As stem cell therapeutics continue to evolve in a wide range of indications, from tissue regeneration, to even curing HIV, the discovery of cancer stem cells (CSCs) and their critical role in enabling solid tumors to spread and recur throughout the body, has led to companies looking to target these cells as a way to treat and prevent metastatic cancer.

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Cancer stem cells are resistant to standard treatments because they can lie dormant for long periods, then migrate to other organs and trigger explosive tumor growth, causing the patient to relapse. Eighty percent of cancers are from solid tumors and metastasis is the main cause of patient death. PRP is designed to target and eradicate cancer stem cells not killed by radiation or chemotherapy. By treating solid tumors with PRP, the tumor loses the ability to generate new cells and the tumor disappears, with no option to form a metastatic tumor elsewhere.

The Company’s scientific researchers have published data confirming that PRP regulates up to 4 relevant pathways, TGFβ, Hippo, Wnt, and Notch, related to cancer spread and metastasis of CSCs. That cascade of reactions disrupts CSC characteristics that leads to tumor invasion into surrounding tissues. PRP interferes with the signals that the primary tumor sends to other tissues to prepare the pre-metastatic niche.

"PRP is a targeted, CSC therapy for the treatment and prevention of metastatic cancer, which I believe can help achieve a total victory in the fight against metastatic cancer from solid tumors," said Dr Julian Kenyon, M.B.Ch.B., M.D., Propanc’s Chief Scientific Officer. "We have been working with cancer stem cells for several years and our preclinical studies demonstrate that PRP has a significant effect on suppressing these cells, which are the main drivers of cancers. I am impressed in the lab, looking down through the microscope at pancreatic CSCs, then adding PRP, and many disappear. That was really remarkable and is likely to put us ahead of the competition. This is highly significant because suppressing CSCs reduces the risk of tumor recurrence, clinically."

Mr. James Nathanielsz, Propanc’s Chief Executive Officer said, "Through scientific research, our discoveries provide evidence that our technology can control the spread of cancer. As a result of international borders reopening since the pandemic, we plan to visit our joint research partners in Spain, as well as undertake a road show in the US, advancing our company’s partnerships as we prepare for a First-In-Human study in advanced cancer patients."

Genprex Announces First Patient Dosed in Phase 1/2 Acclaim-1 Clinical Trial of
REQORSA™ Immunogene Therapy in Combination with Tagrisso® to Treat
Non-Small Cell Lung Cancer

On March 2, 2022 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the first patient was dosed in the Acclaim-1 clinical trial, an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA Immunogene Therapy, in combination with Tagrisso (osimertinib) in patients with late-stage non-small cell lung cancer (NSCLC) whose disease progressed after treatment with Tagrisso (Press release, Genprex, MAR 2, 2022, View Source [SID1234609364]). In 2020, Genprex received U.S. Food and Drug Adminstration’s (FDA) Fast Track Designation for treatment of the Acclaim-1 patient population.

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"This is an exciting milestone for Genprex that we believe brings us one step closer to achieving our goal to validate the potential benefits of REQORSA in NSCLC patients who have life-limiting disease and few treatment options," stated Mark S. Berger, M.D., Chief Medical Officer of Genprex. "Based on both the preclinical data and previous clinical data, REQORSA has the potential to improve the response to current targeted therapies, such as Tagrisso, for this study patient population. We look forward to continuing to advance this important trial."

The Acclaim-1 clinical trial is an open-label, multi-center Phase 1/2 clinical trial evaluating the Company’s lead drug candidate, REQORSA, in combination with Tagrisso in patients with late-stage NSCLC with activating epidermal growth factor receptor ("EGFR") mutations whose disease progressed after treatment with Tagrisso. Genprex expects the Phase 1 portion of the Acclaim-1 trial to enroll up to 18 patients in a dose escalation study to determine the maximum tolerated dose of the combination. The Phase 2 portion of the study is expected to enroll approximately 74 patients to be randomized 1:1 to receive either REQORSA and Tagrisso combination therapy or Tagrisso monotherapy. The primary endpoint of the Phase 2 portion of the trial is progression-free survival, which is defined as time from randomization to progression or death. An interim analysis will be performed at 25 events.

Genprex recently added four new clinical trial sites for the Acclaim-1 trial in order to accelerate enrollment, bringing the current number of sites to five. The Company expects to add additional sites for the Phase 2 portion of the trial.

Genprex previously announced the Centralized Institutional Review Board (IRB) approval for the Acclaim-1 clinical trial in NSCLC. Additional information about the Acclaim-1 clinical trial can be found by visiting ClinicalTrials.gov.

"Despite recent advances in immuno-oncology and other targeted treatments, NSCLC remains a significant unmet medical need as resistance to Tagrisso and other targeted therapies is a common clinical problem," said Dr. Berger. "REQORSA is a pan-kinase inhibitor shown to inhibit both the EGFR and AKT oncogenic kinase pathways. We believe that REQORSA’s multimodal activity will block emerging bypass pathways, reducing the probability that drug resistance develops."

Preclinical Data at AACR (Free AACR Whitepaper) 2021

Supportive preclinical data on the combination of REQORSA and Tagrisso (osimertinib) were presented by Genprex collaborators at the 2021 American Association of Clinical Research (AACR) (Free AACR Whitepaper) annual meeting. The data showed that REQORSA and Tagrisso had a synergistic antitumor effect in EGFR mutant Tagrisso resistant NSCLC tumor xenograft models. In the preclinical study, researchers developed an osimertinib-resistant H1975-OsiR isogenic cell line through continuous exposure to osimertinib. Xenograft tumors from both H1975-parental and H1975-OsiR cells were grown in NSG mice and were treated with osimertinib. The combination of REQORSA and osimertinib showed a robust antitumor effect compared with single agent treatment groups against H1975-OsiR tumors.

Tagrisso is a registered trademark of AstraZeneca plc and its largest selling drug with 2021 sales of over $5 billion.

About REQORSA

REQORSA Immunogene Therapy (quaratusugene ozeplasmid) for non-small cell lung cancer (NSCLC) uses Genprex’s unique, proprietary ONCOPREX Nanoparticle Delivery System, which is the first systemic gene therapy delivery platform used for cancer in human clinical trials.

The active ingredient in REQORSA is the TUSC2 gene, a tumor suppressor gene. REQORSA consists of the TUSC2 gene encapsulated in a nanoparticle made from lipid molecules with a net positive electrical charge. REQORSA is injected intravenously and can specifically target cancer cells, which generally have a negative electrical charge. Once REQORSA is taken up into a cancer cell, the TUSC2 gene is expressed, and the TUSC2 protein is capable of restoring certain defective functions arising in the cancer cell. REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for programmed cell death, or apoptosis, in cancer cells, and modulates the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance.

Black Diamond Therapeutics to Participate at the Cowen 42nd Annual Health Care Conference

On March 2, 2022 Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a precision oncology medicine company pioneering the discovery and development of MasterKey therapies, reported that its President and Chief Executive Officer, David M. Epstein, Ph.D., will participate in a panel discussion on lung cancer at the Cowen 42nd Annual Health Care Conference being held virtually on Wednesday, March 9, 2022 at 9:10 a.m. ET (Press release, Black Diamond Therapeutics, MAR 2, 2022, View Source [SID1234609363]).

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A live webcast of the panel discussion can be accessed by visiting the investor relations section of the Company’s website, www.blackdiamondtherapeutics.com. A replay of the panel discussion will also be available and archived on the site for 30 days.

Mersana Therapeutics to Present at the Cowen 42nd Annual Health Care Conference

On March 2, 2022 Mersana Therapeutics, Inc., (NASDAQ: MRSN) a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, reported that the Company will participate in a virtual panel presentation at the Cowen 42nd Annual Health Care Conference on Wednesday, March 9, 2022 at 12:50 p.m. ET (Press release, Mersana Therapeutics, MAR 2, 2022, https://ir.mersana.com/news-releases/news-release-details/mersana-therapeutics-present-cowen-42nd-annual-health-care [SID1234609362]).

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A live webcast of the presentation will be available on the Investors & Media section of Mersana’s website at www.mersana.com. An archived replay will be available for approximately 30 days following the presentation.