On June 11, 2020 CNS Pharmaceuticals, Inc., (Nasdaq: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its lead product Berubicin for the treatment of malignant gliomas (Press release, CNS Pharmaceuticals, JUN 11, 2020, View Source [SID1234561018]).

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"We are pleased to receive Orphan Drug Designation for Berubicin, our lead candidate. The designation provides Berubicin with a special status that can accelerate its development to treat malignant gliomas, and provides CNS with the potential for market exclusivity upon the drug’s approval," stated John Climaco, CEO of CNS Pharmaceuticals. "In the Phase 1 trial of Berubicin to treat glioblastoma, one of the world’s most aggressive cancers, under a prior developer, 44% of the patients demonstrated a significant improvement in progression free survival, and one patient experienced a complete response. We look forward to continuing to execute on our strategic plan and initiating a Phase II trial evaluating the effect of Berubicin on patients with glioblastoma later this year."

Chief Medical Officer of CNS, Dr. Sandra Silberman, stated, "We are excited to continue to drive the development of Berubicin and work towards addressing a critical unmet medical need. Glioblastoma currently has a dismal survival rate of only 14.6 months from its diagnosis. We believe Berubicin, which based on limited clinical data appears to be the first anthracycline to cross over the blood brain barrier in adults, provides a potentially novel therapy for the treatment of malignant gliomas."

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at any given time. Due to small patient numbers, treatment for these rare diseases would not be considered economically feasible without government programs to support their economic viability. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

About Berubicin

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata.

On June 11, 2020 AVM Biotechnology reported that it has been awarded a National Cancer Institute Small Business Innovation Research (SBIR) Grant from the National Institutes of Health (NIH) to study the use of their lead molecule AVM0703 as a preconditioning agent to allow safe and efficient delivery of therapeutic immune cells for cancer treatment (Press release, AVM Biotechnology, JUN 11, 2020, View Source [SID1234561017]). This novel solution could offer clinical advantages to any cell-based immunotherapy, improving access to potentially life-saving therapies by all cancer patients, including those too frail to receive chemotherapy.

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Cellular immunotherapy has the potential to become a crucial solution for cancer. However, toxic chemotherapy is currently required as preconditioning treatment to impair graft rejection and maintain therapeutic cells in the bloodstream where they can target cancer cells. Chemotherapy is hardly tolerated by frail cancer patients, and it fuels the side effects of immunotherapy such as toxic cytokine releases (CRS) and neuroedemas. AVM0703, which could be easily administered to increase efficient delivery of adoptive cellular immunotherapy, induces safe lymphodepletion in only 24 hours, sparing platelets, stem cells and red blood cells in animal models. AVM0703 can safely deplete monocytes, known to be a key inducer of CRS. CRS toxicities occur as frequently as 90% with half of them determined as severe. Severe CRS complications can be life threatening if not treated in a timely manner. Unlike chemotherapy, AVM0703 could safely deplete monocytes reducing the risk of CRS and making cellular immunotherapy accessible to high-risk individuals.

This Phase I grant will be used to validate the efficacy and safety of preconditioning by AVM0703 in an established tumor mouse models of multiple myeloma (MM). The proposal was regarded as very significant in addressing a clinical need of better ways for improving efficacy and reducing toxicity of cellular therapies. Moreover, preconditioning using AVM0703 was seen as well supported by a good rationale and strong preliminary data. AVM0703 mode of action could offer an exemplary preconditioning regimen.

Named one of the top 10 best Biotech and Pharma companies to keep your eye on in 2019 by Mirror Review Online Magazine, AVM Biotechnology was founded in 2008 by Dr. Theresa Deisher, Ph.D. With over 30 years of successful pharmaceutical research experience and holding over 47 patents, Dr. Deisher leads a team of scientists dedicated to changing what a diagnosis of cancer, autoimmunity, or chronic infectious disease means to patients and their loved ones. AVM received FDA IND approval in April 2020 to test AVM0703 for treatment of relapsed/refractory lymphoid malignancies. AVM’s passion is to deliver drugs that work rapidly and that are safe, effective, and affordable, to treat serious worldwide illness like cancer, autoimmunity, and life-altering infectious disease. They develop products that improve outcomes without additional suffering because they believe that side effects from treatments of cancers or infections should never be worse than the diseases themselves.

AVM is the only company to receive homologous use designation for a patient’s own bone marrow used for an indication outside of blood disorders. Additionally, in 2019 AVM Biotechnology was awarded an SBIR grant for targeted lympho-ablation as an alternative to cure Type I Diabetes by the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK).

This award was granted by the National Institutes of Health under Award Number R43CA246896. The content of this press release is solely the responsibility of the author and does not necessarily represent the official views of the NIH.

On June 11, 2020 Accellix, the Cell Therapy QC company, reported a Series D financing round of $9.5 million led by bioMérieux, a world leader in the field of in vitro diagnostics for over 55 years (Press release, Accellix, JUN 11, 2020, View Source [SID1234561016]). All existing major investors participated in this raise and have also agreed to convert their pre-existing convertible note in the amount of $8.5 million, bringing the company’s total funding in this round to $18 million. This latest round of funding will be used to further expand the market reach of the Accellix system for Cell Therapy QC, enabling its customers greater access to the expertise, scale, resources, and technology needed to effectively assess the quality of life saving immunotherapies.

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"Companies developing cellular therapies in the area of immuno-oncology and other therapeutic areas understand the need to replace costly and complicated flow cytometry based procedures with an easy to use sample-to-answer system. Being recognized by bioMérieux for our achievements to-date and having access to their vast array of resources and expertise, will help us serve our existing customers while attracting new ones," said Nir Nimrodi, Accellix Chairman and CEO.

"The Accellix platform eliminates extensive training and complex operational procedures by automating inherent manual complex flow cytometry processes. It provides accurate and immediate flow cytometry results at the point of need, removing the requirement of sending samples out to a central lab. We have been searching for such a platform that will complement our Industry-leading Microbial Quality Control offering and it is our intention to work with Accellix on enabling them to advance their market reach and service and support capabilities," stated Michael Reynier, bioMérieux Vice President, Healthcare Industries Business Area. "Testing a precious cell therapy product for sterility and for quality, swiftly, accurately and reproducibly, will improve patients’ access to the lifesaving cellular therapies that are currently being developed."

On June 11, 2020 City of Hope, a world-renowned independent research and treatment center for cancer, diabetes and other life-threatening diseases, reported that it has signed an exclusive, worldwide licensing agreement with Scopus BioPharma Inc., a biopharmaceutical company focused on novel therapeutics for serious diseases with significant unmet medical needs (Press release, City of Hope, JUN 11, 2020, View Source [SID1234561015]). Scopus will further develop and plans to commercialize a City of Hope first-in-class, targeted immuno-oncology gene therapy.

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A first in-human phase 1 clinical trial for B cell lymphoma patients that uses the licensed gene therapy drug, CpG-STAT3siRNA, a STAT3 inhibitor, is expected to commence at City of Hope in the second half of this year.

Growing evidence links B cell non-Hodgkin lymphomas to persistent activation of STAT3, a gene that drives tumor cell growth and anti-tumor immune suppression. The STAT3 inhibitor is a highly selective and targeted therapy that silences the activity of the STAT3 gene by way of RNA interference. It also stimulates the TLR9 receptors to activate the body’s immune defense to recognize and kill cancer cells.

In preclinical testing at City of Hope, the STAT3 inhibitor has successfully reduced growth and metastasis of various preclinical tumor models, including melanoma, and colon and bladder cancers, as well as leukemia and lymphoma.

City of Hope’s Hua Yu, Ph.D., Billy and Audrey L. Wilder Professor in Tumor Immunotherapy, associate chair/professor in the Department of Immuno-Oncology, and co-leader of the Cancer Immunotherapeutics Program, and Marcin Kortylewski, Ph.D., associate professor in the Department of Immuno-Oncology, who are both leading experts in the role of STAT3 in tumor angiogenesis and tumor immune evasion and in oligonucleotide-based cancer immunotherapies, developed the STAT3 inhibitor. The strategy was developed based on seminal discoveries by Yu’s team defining the key role of STAT3 in cancer cell survival and immune tolerance, combined with pioneering work by Kortylewski’s team on STAT3 targeting using TLR9-targeted delivery of siRNA oligonucleotide therapeutics into immune cells.

"City of Hope is proud to work with Scopus to bring this long-anticipated STAT3 drug to our clinical trial patients," Yu said. "STAT3 is critical for the survival and metastasis of cancer cells, and for suppressing anti-tumor immune responses. If City of Hope and Scopus can develop the first therapy against STAT3, many cancer patients will benefit from this lifesaving drug."

"Our laboratories were the first to demonstrate that successful cancer immunotherapy needs to be two-step since TLR9 immunostimulation is only effective when STAT3 in the tumor microenvironment is no longer active," Kortylewski added. "It is exciting to see this technology approaching clinical application with a strong ally in biopharma."

Both an academic medical center and a drug development powerhouse, City of Hope is known for creating the technology used in the development of human synthetic insulin and numerous breakthrough cancer drugs. Its unique research and development hybrid of the academic and commercial creates an infrastructure that enables City of Hope researchers to submit an average of 50 investigational new drug applications to the U.S. Food and Drug Administration each year. The institution currently holds more than 450 patent families.

"Scopus is extremely excited about licensing such a promising gene therapy drug with a robust intellectual property portfolio, compelling preclinical efficacy and safety profile, and a plan to enter phase 1 this year," said Joshua R. Lamstein, Scopus co-chairman. "We are privileged to have the opportunity to work with City of Hope, a globally recognized research institution and cancer center. We are looking forward to working with Drs. Yu and Kortylewski, who are among the preeminent researchers in immuno-oncology."

On June 11, 2020 Egle Therapeutics SAS, an emerging biotechnology company focused on developing first-in-class immunotherapies targeting immune suppressor regulatory T-cells for oncology and autoimmune diseases, reported a corporate strategic research alliance with Takeda Pharmaceutical Company Limited ("Takeda") with the goal of validating novel tumor-infiltrating regulatory T-cell (Treg) targets against which Takeda will develop potential therapies (Press release, Egle Therapeutics, JUN 11, 2020, View Source [SID1234561014]).

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As a newly established bioscience company spun out of Institut Curie in the field of Treg immune modulation, Egle Therapeutics has assembled a proprietary translational based target discovery engine leveraging patient samples to map out – at the single cell level – unique transcriptomic signatures and targets specific for tumor-infiltrating Treg sub-populations. Capitalizing on these newly identified tumor-infiltrating Treg targets, the company is building a proprietary Treg modulating drug pipeline which also includes computationally designed resurfaced IL-2 proprietary variants, featuring unique mechanism of action to engage or dis-engage Tregs.

Under the terms of the agreement, Egle Therapeutics will lead target validation efforts on a subset targets Egle has identified and Takeda will be responsible for the development, manufacturing, and commercialization of resulting therapies. Egle Therapeutics will receive an upfront payment and research funding, and will be eligible to receive additional development and sales milestone payments based on the exclusivity of targets selected by Takeda.

Luc Boblet, co-founder and CEO of Egle Tx, commented "We are thrilled to engage with Takeda to fully exploit the great therapeutic potential of inhibiting the most immunosuppressive arm of immunity to restore antitumor immune response. We believe that joining forces would be the most efficient path to successfully develop novel generation of anti-Treg immunotherapies for the benefit of patients."

"Working with Egle to leverage the unique translational derived patient data bringing tumor-specific Treg targets is an exciting prospect to further advance our immuno-oncology drug discovery efforts," said Loïc Vincent, Head, Oncology Drug Discovery Unit at Takeda. "Targeting tumor-infiltrating regulatory T-cells to overcome the immune suppression in tumor microenvironments will complement our current immuno-oncology approaches and help advance an exciting portfolio."

In addition to the funding of the multi-target research collaboration, Takeda will invest €4.6 million in convertible debt through its venture arm, Takeda Ventures, Inc. ("TVI"), to support Egle’s corporate growth and internal programs on IL-2-based Treg modulation. Egle’s flagship program pioneers the dis-engagement of tumor-infiltrating Treg through unique proprietary series of IL-2 variants that act as IL-2 antagonists.

"While the whole competition converges solely on a similar approach to enhance effector T cells by disrupting binding of IL-2 to Tregs, our IL-2 variants conserve intact IL-2 binding to CD25, conferring a unique mechanism of action for starving Treg from endogenous IL-2, while leaving active the CD8+ effector T cell arm of the immune response," Luc Boblet added. "Takeda’s equity commitment will help Egle’s launch strikingly hit the road building on our excellent scientific foundation."