On January 4, 2022 ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, reported that it has been granted a U.S. patent (11,207,350 B2) for a novel natural killer (NK) cell therapy composition and method for treating cancer that combines the company’s genetically modified NK-92 cells with CD16 receptors to enhance binding and activity of monoclonal antibodies (Press release, ImmunityBio, JAN 4, 2022, View Source [SID1234598077]). The combination of these engineered NK-92 cells with current monoclonal antibody therapies has the potential to augment the overall cytotoxic effects of monoclonal antibody treatment alone and help to address relapse by bolstering the patient’s own natural immune system response.

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ImmunityBio’s NK-92 cells are specifically modified versions of the NK cells that are a core element of the human immune system. First discovered in the blood of a non-Hodgkins lymphoma patient, these cells retain most of the activating receptors of NK cells but lack the major inhibitory receptors, giving them higher baseline cytotoxicity against tumor cells. The cells have been included in more than 450 published papers covering a wide range of research and they are currently being studied in clinical trials for pancreatic cancer, triple-negative breast cancer, and non-Hodgkin lymphoma.

The off-the-shelf NK cell platform can be easily expanded, genetically modified and cryopreserved. By inserting chimeric antigen receptors (CARs) in the NK-92 cells, they have the potential for multi-specific killing including direct NK cell killing via stress ligands, CD16-mediated killing via tumor-targeted antibodies, and CAR-mediated killing.

The patent, which protects this therapeutic approach for 20 years from its original filing, covers the use of these NK-92 cells in conjunction with the mAbs that are a standard of care in many cancer cases.

"While monoclonal antibodies have significantly improved the clinical outcomes in patients with cancer, many of these patients ultimately relapse," said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "A growing body of research suggests that clinically meaningful responses to these antibody therapies is dependent upon the overall health of the patient’s own natural killer cells and whether the cells express the high-affinity variant of the CD16 receptors, which are found on the surface of natural killer cells. Only about 10% of NK cells in the blood of healthy people have the high-affinity CD16 receptors for monoclonal antibody binding—the majority of NK cells have the intermediate- and low-affinity receptor—therefore an infusion of these NK-92 cells engineered with a high-affinity CD16 receptor supercharges the monoclonal antibodies, potentially enabling the antibodies to be therapeutically more effective in combination with the killing activity of these engineered NK-92 cells."

The new patent adds another asset to ImmunityBio’s strong intellectual property portfolio, which includes more than 1,100 issued and pending patents worldwide across multiple categories including biologics, vaccine vectors, natural killer cells, and GMP devices. Patents for key areas such as N-803 (Anktiva), adenovirus vaccine vectors, yeast vaccine vectors, NK-92 cells and therapies extend to 2036 and beyond.

On January 4, 2022 Immix Biopharma reported that The FDA has granted a rare pediatric disease designation (RPDD) to IMX-110 for the treatment of rhabdomyosarcoma, according to an announcement from Immix Biopharma, Inc., the drug developer (Press release, Immix Biopharma, JAN 4, 2022, View Source [SID1234598076]).1

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The novel product is a Tissue-Specific Therapeutic that was built on the clinical-stage biopharmaceutical company’s TME Normalization Technology, which allows for the agent to circulate in the bloodstream, subsequently exit through porous tumor blood vessels, and accumulate in the tumor microenvironment.2

Then, IMX-110 simultaneously attacks the following 3 components of the microenvironment: cancer-associated fibroblasts, tumor-associated macrophages/immune cells, and the cancer itself; this severs the lifelines between the tumor and its metabolic and structural support. The product’s technology causes tumor apoptosis.

RPDD is only given to products developed to treat serious and life-threatening diseases that primarily affect children aged up to 18 years of age, and impact fewer than 200,000 individuals in the United States. If a new drug application for the agent is approved in the country, the company may be eligible to receive a priority review voucher from the agency, which can then be redeemed to obtain priority review for any subsequent marketing application.

"We are pleased by the FDA’s acknowledgement of the urgent need for a safe and effective treatment for children with this devastating disease," Ilya Rachman, MD, PhD, chief executive officer of Immix Biopharma, Inc., stated in a press release.

Clinical findings across several soft tissue sarcoma subtypes in several heavily pretreated patients indicated that the investigational agent resulted in a median progression-free survival (PFS) of 4.0 months.3 Tumor shrinkage was observed in 75% of these patients. Moreover, no treatment-related serious adverse effects (AEs), nor dose interruptions due to toxicity, were reported.

"We are encouraged by our phase 1b/2a clinical data in soft tissue sarcoma," Rachman added. "IMX-110 is a tissue-specific therapeutic that simultaneously attacks all 3 components of the tumor microenvironment, severing the critical lifelines between the tumor and its metabolic and structural support. We believe our SMARxT platform generating Tissue-Specific Therapeutics represents a distinct alternative to the traditional ‘single target, single mutation’ development model."

The safety, tolerability, and pharmacokinetics of IMX-110 in patients with advanced solid tumors is currently under investigation as part of an open-label, multicenter, dose-escalation/-expansion phase 1/2 trial (NCT033892340).4

To be eligible for enrollment, patients need to be at least 18 years of age, have a confirmed advanced solid tumor as per histology and have progressed or are refractory or intolerant to standard treatment. Patients need to have an ECOG performance status ranging from 0 to 2, a life expectancy of at least 3 months, acceptable cardiac function, and must meet certain laboratory requirements.

If patients have a history of severe allergic reactions to any unknown allergens or any components of the study drug formulation, received chemotherapy within 14 days of dosing, immunotherapy within 28 days of dosing, or a biologic of hormonal therapy within 28 days of dosing, they will be excluded.

Other exclusion criteria include having participated in any other drug study up to 4 weeks before study drug administration, requiring surgery or benefit from other anticancer agents, a history of and/or risk factors for ischemic heart disease, no recovery of toxicities from prior treatment, and being positive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus.

The primary outcome measures are examining the number of treatment-related AEs and identifying the maximum tolerated dose of IMX-110 and the recommended phase 2 dose of the agent. Secondary outcome measures include evaluating plasma concentrations of IMX-110, response rate, PFS, overall survival, and duration of response.

Investigators will also examine the pharmacodynamic activity of the agent with appropriate biomarkers.

Previously, in September 2021, the FDA granted an orphan drug designation to IMX-110 for use as a potential therapeutic in patients with soft tissue sarcoma.2

On January 4, 2022 iBio, Inc. (NYSEA:IBIO) ("iBio" or the "Company"), a developer of next-generation biopharmaceuticals and pioneer of the sustainable, plant-based FastPharming Manufacturing System, reported that it will participate in the H.C. Wainwright BioConnect Conference, to be held virtually January 10-13, 2022 (Press release, iBioPharma, JAN 4, 2022, View Source [SID1234598075]).

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iBio’s pre-recorded presentation will be available on demand during the event for all registered attendees. Beginning on Monday, January 10, 2022, the presentation will also be available on the Company’s website at www.ibioinc.com under "News & Events" in the Investors section.

On January 4, 2022 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported that the Company will present at the virtual H.C. Wainwright BioConnect Conference, January 10 – 13, 2022 (Press release, Hookipa Biotech, JAN 4, 2022, https://ir.hookipapharma.com/news-releases/news-release-details/hookipa-pharma-present-hc-wainwright-bioconnect-conference [SID1234598074]). The presentation will be available beginning at 7:00 AM EST on Monday, January 10.

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The webcast of the presentation will be available within the Investors & Media section of HOOKIPA’s website at View Source An archived replay will be accessible for 30 days following the event.

On January 4, 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 it has expanded its oncology research and development pipeline to include small cell lung cancer (SCLC) as an additional disease indication for its lead drug candidate, REQORSA Immunogene Therapy (Press release, Genprex, JAN 4, 2022, View Source [SID1234598073]). SCLC represents approximately 10-15 percent of the lung cancer market, while REQORSA’s initial target indication of non-small cell lung cancer (NSCLC) represents approximately 84 percent of the lung cancer market.

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"Data from human studies indicate that REQORSA could be beneficial in targeting small cell lung cancer," said Mark S. Berger, MD, Chief Medical Officer at Genprex. "Like non-small cell lung cancer, small cell lung cancer consistently has low TUSC2 protein levels and is documented to often have deletion of one TUSC2 gene allele. Extensive stage small cell lung cancer has a very poor prognosis, with a median progression free survival of 5.2 months. Expanding the therapeutic indications targeted by REQORSA to include small cell lung cancer may provide us with another important clinical opportunity to combine REQORSA with small cell lung cancer therapies, including checkpoint inhibitors."

REQORSA consists of a TUSC2 gene expressing plasmid encapsulated in non-viral lipid nanoparticles. Genprex’s oncology program utilizes its unique, proprietary, non-viral ONCOPREX Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. The ONCOPREX Nanoparticle Delivery System could allow for the delivery of a number of cancer-fighting genes, alone or in combination with other cancer therapies to potentially address unmet medical need in cancer, thereby potentially improving patient outcomes through the advancement of multiple therapeutic approaches for large patient populations.

"REQORSA may have the potential to treat small cell lung cancer, in addition to non-small cell lung cancer, thus making REQORSA a possible drug candidate for the entire lung cancer market," said Rodney Varner, Genprex’s Chief Executive Officer. "Lung cancer continues to be the leading cause of cancer deaths worldwide, causing more deaths than colorectal, breast, liver or stomach cancers. We hope that one day REQORSA will provide improved outcomes for virtually all lung cancer patients."