On April 6, 2022 CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, reported it has received approval from the National Agency for the Safety of Medicine and Health Products (ANSM) Competent Authority and from the People Protection Ethics Committee (EC) SUD-EST III (CPP Sud-Est III) in France for the Company’s potentially pivotal study of Berubicin for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer (Press release, CNS Pharmaceuticals, APR 6, 2022, View Source [SID1234611554]).

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"Access to patients is the lifeblood of any clinical study and this approval, in the second most populous country in Europe, provides just that for our Berubicin trial. We have said time and again that our number one priority is the advancement of this potentially pivotal study. This is evidenced by our continuous dedication to driving enrollment and bringing global clinical sites on line. We are grateful to the French Competent Authority and Ethics Committee and their positive feedback on what we believe is an incredibly important clinical program. Across the globe there is an urgent need for GBM treatment options. We will continue to press on in our efforts to advance Berubicin through the clinic and importantly, to patients and their families. I am proud of the progress our team has made to-date and believe there are additional clinical sites to join those that have already been added to this globlal potentially pivotal study," commented John Climaco, CEO of CNS Pharmaceuticals.

Dr. Carole Gourmelon, MD, Institut de Cancérologie de l’Ouest, St Herblain, added, "GBM is a devastating disease with significant unmet need. I have been encouraged by the data Berubicin has demonstrated to date and look forward to further evaluating its potential to provide benefit to patients. Now with the necessary approvals received, I look forward to joining the Company’s efforts to progress Berubicin through the clinic."

Berubicin is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier currently being evaluated in a potentially pivotal global study evaluating its efficacy and safety in the treatment of GBM. The potentially pivotal global trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy. Approximately 243 patients with GBM after failure of standard first line therapy will be randomized in a 2:1 ratio to receive Berubicin or lomustine for the evaluation of Overall Survival, the primary endpoint of the study. Overall Survival is a rigorous endpoint that the U.S. Food and Drug Administration (FDA) has recognized as a basis for approval of oncology drugs when a statistically significant improvement can be shown relative to a randomized control arm.

A pre-planned, non-binding futility analysis will be performed after approximately 30 to 50% of all planned patients have completed the primary endpoint at 6 months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis.

The FDA recently granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with the FDA to expedite the development and review process. As previously announced, the Company also received Orphan Drug Designation from the FDA which may provide seven years of marketing exclusivity upon approval of an NDA.

For more information about the potentially pivotal Berubicin trial, visit clinicaltrials.gov and reference identifier NCT04762069.

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 Pharmaceuticals, Inc. Berubicin, was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.

On April 6, 2022 AffyImmune Therapeutics, Inc., a clinical stage biotechnology company using its Tune & Track platform to develop CAR T cells for the treatment of solid cancers, reported that it will present an abstract in a poster session at the 2022 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in New Orleans, Louisiana, which is being held April 8 – 13 (Press release, AffyImmune Therapeutics, APR 6, 2022, View Source [SID1234611550]). Presented findings will highlight research using AffyImmune’s Tune & Track platform for the affinity tuning of the interaction between CAR T cells and cancer antigen, tracking of CAR T cells in real-time, and armoring of CAR T cells with cytokines.

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Details of the abstract and poster are as follows:

Title: Focused IL-12 cytokine delivery enhances function of affinity-tuned and real-time tracked ICAM-1-specific CAR T cells in solid tumors
Presenting Author: Michael Gallagher, PhD, Scientist
Session Category: Adoptive Cell Therapy 1
Poster Number: 558/12
Abstract Number: 4978
Presentation Type: Poster
Date, Time, and Location: Sunday, April 10, 1:30 – 5:00 pm, Poster Section 36

On April 6, 2022 Lunit reported the presentation of two abstracts featuring its AI research on cancer treatment at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 (Press release, Lunit, APR 6, 2022, View Source [SID1234611545]). The meeting will be held from April 8 to 13 at the Ernest N. Morial Convention Center in New Orleans, Louisiana. This year marks Lunit’s fourth time presenting its findings at AACR (Free AACR Whitepaper).

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As a leading provider of state-of-the-art cancer diagnostic technology, Lunit has focused on developing novel AI biomarkers for application in immunotherapy. Since 2019, the company has released groundbreaking findings based on its AI-powered tissue analysis platform, ‘Lunit SCOPE,’ demonstrating the software’s predictive value in identifying patients eligible for immunotherapy. The upcoming AACR (Free AACR Whitepaper) presentations will showcase Lunit’s AI biomarker platform, Lunit SCOPE IO—part of the Lunit SCOPE product line.

Lunit SCOPE IO analyzes a patient’s cancer tissue slide image by observing the distribution of tumor-infiltrating lymphocytes (TIL)—one of the representative immunocytes that fight cancer cells. Based on the spatial distribution pattern of TILs and cancer cells in the tumor microenvironment, Lunit SCOPE IO identifies the tissue sample as one of three immune phenotypes: inflamed, immune-excluded, and immune-desert.

In one of its abstracts, Lunit presents the correlation between its AI-based immune phenotype method and aberrant transforming growth factor-beta (TGF-B) pathways in pan-carcinoma.

For TILs to fight cancer cells, they must be located in proximity to each other. The aberrant TGF-B pathway in the tumor environment has been highlighted as one of the core resistance pathways that inhibit immunotherapy by excluding TILs from the tumor area. This type of distribution is classified as the immune-excluded phenotype, in which TILs are separated from the cancer cells.

Upon conducting a large-scale, pan-carcinoma analysis, Lunit’s research team found that an aberrant TGF-B pathway is indeed associated with the immune-excluded phenotype, as well as increased proportions of cancer stroma.

"This was a multi-omics study incorporating genome-based analysis that shows a direct correlation between immune phenotypes with the TGF-B pathway," said Chan-Young Ock, Chief Medical Officer at Lunit. "By developing our AI to analyze TIL and their spatial relationship with cancer stroma, Lunit was able to initiate the first study that directly compares immune phenotypes and TGF-B expression on a large-scale database."

The company will also deliver an oral presentation on the distinct clinical outcomes and molecular profiles among immune phenotypes in endometrial cancer.

Lunit’s studies thus far first focused on the validity of immune phenotyping as a biomarker for advanced non-small cell lung cancer (NSCLC). However, this study demonstrates that similar AI-based spatial analysis can bring clinically significant results as a biomarker in endometrial cancer.

The study aimed to analyze the differences between the three immune phenotypes in endometrial cancer using clinical data, pathological slides, and genetic expression registered in the National Cancer Institute’s "The Cancer Genome Atlas" (TCGA). Results indicated that the inflamed phenotype (TILs located close to cancer cells) showed the best overall survival outcome. Furthermore, this particular phenotype was associated with the highest expressions of PD-L1 and CTLA4—immune checkpoint proteins that act as important biomarkers in predicting immunotherapy response.

"Given the significant differences in survival outcome depending on each phenotype, AI-based tumor microenvironment classification using Lunit SCOPE IO may serve as a clinically significant, prognostic biomarker in endometrial cancer," said Brandon Suh, CEO of Lunit. "We plan to expand the range of our research to validate the efficacy of Lunit SCOPE IO in all cancer types originating from the epithelium."

The Lunit team will be exhibiting at AACR (Free AACR Whitepaper), at booth 1364.

On April 6, 2022 Biocytogen subsidiary Eucure Biopharma reported the first patient dosing for a phase I clinical trial (No. YH003005) of YH003 (anti-CD40 monoclonal antibody, mAb) in combination with YH001 (anti-CTLA-4 mAb) and pembrolizumab (anti-PD-1 mAb) in Australia (Press release, Eucure, APR 6, 2022, View Source [SID1234611543]).

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The study is an open-label, dose-escalation study designed to evaluate the safety, tolerability and efficacy of YH003 in combination with YH001 and pembrolizumab in patients with advanced solid tumors. Pharmacokinetics and immunogenicity of YH003 will also be evaluated.

"Previous phase I clinical trials of YH003/Toripalimab (anti-PD-1 mAb) combination therapy and YH001/Toripalimab combination therapy indicate desirable safety profiles and preliminary efficacy for both products," said Dr. Yuelei Shen, Chairman and CEO of Biocytogen and Eucure Biopharma. "The combination of CTLA-4, PD-1 and CD40 mAbs is based on their different but complementary biological mechanisms; we hope that the three-drug combination study can further strengthen the antitumor efficacy to benefit patients."

About YH003

YH003 is a humanized IgG2 agonistic CD40 antibody. Whether used as a single agent or in combination with anti-PD-1 mAb drugs, YH003 demonstrated strong anti-tumor effects against multiple tumor models in Biocytogen’s humanized CD40 mice, without exhibiting hepatotoxicity or other toxicities. Pharmacodynamic studies in mice indicate that YH003 significantly increased the infiltration of anti-tumor T cells into tumors.

About YH001

YH001 is an anti-CTLA-4 monoclonal antibody. CTLA-4 is a key target for tumor immunotherapies, due to the potential to enhance the immune response to tumor cells and promote removal of regulatory T cells (Treg) from the tumor microenvironment. Blocking the inhibitory signals from both CTLA-4 and PD-1 to enhance the anti-tumor responses is considered a promising tumor immunotherapy, as they control the different types of T cells.

On April 6, 2022 Everest Medicines (HKEX 1952.HK, "Everest", or the "Company"), a biopharmaceutical company focused on developing and commercializing transformative pharmaceutical products to address critical unmet needs in Asia Pacific markets, reported that it has entered into a memorandum of understanding for a partnership with China Resources Pharmaceutical Group Limited (HKEX 3320.HK, "CR Pharma") with the intent to establish an independent company ("the mRNA Co.") focused on the discovery, development and commercialization of messenger RNA ("mRNA") vaccines (Press release, Everest Medicines, APR 6, 2022, View Source [SID1234611541]).

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CR Pharma is a subsidiary of China Resources (Holdings) Co., Ltd, a key state-owned enterprise overseen by China’s State-owned Assets Supervision and Administration Commission of the State Council (SASAC). CR Pharma is an integrated pharmaceutical company in China, engaging in the R&D, manufacturing, and distribution of pharmaceutical products. Their products include chemical drugs, traditional Chinese medicine, biological drugs and supplements, which cover a wide range of treatment fields, including the cardiovascular system, digestive tracts, metabolism, large-volume intravenous infusion, pediatrics, the respiratory system, Coagulation disorders and immune diseases, etc.

Through this proposed partnership with CR Pharma, the mRNA Co. will be well-positioned to advance its potentially best-in-class mRNA vaccine candidates through Chinese regulatory pathways and into commercialization. Under the terms of the MOU, the mRNA Co. will be a fully functional, independent operating company, by assuming the rights under the existing collaboration with Providence Therapeutics Holdings Inc. ("Providence"), including the full technology platform, as well as Everest’s mRNA manufacturing infrastructure. Everest will be the majority and controlling shareholder of the mRNA Co.

The mRNA Co. will accelerate the late-stage development and registration of its potentially best-in-class mRNA COVID-19 vaccine candidate, PTX-COVID19-B, and continue the development of a second-generation COVID-19 vaccine with broad spectrum activity designed to be effective against but not limited to the Omicron variants, as well as two Collaboration Project with Providence that target new mRNA based vaccines. The mRNA Co. will also continue to advance the construction of Everest’s global GMP manufacturing site in Jiashan, Zhejiang Province, which is expected to be operational by the end of 2022. Once complete, the first phase of manufacturing will be dedicated to PTX-COVID19-B, with an expected annual capacity of 700-800 million doses.

CR Pharma comments that through this cooperation with Everest Medicines, the two companies intend to work together in the development of mRNA COVID-19 vaccine and the development of other potential products using the mRNA technology platform, so as to contribute to China’s public health.

"We are pleased with Everest’s ability to continually grow the business, as well as its industry leadership and reputation, by executing strategic collaborations and partnerships with key stakeholders like CR Pharma, which provide valuable expertise and resources to critical ventures such as this," said Wei Fu, Chairman of Everest Medicines and Chief Executive Officer of CBC Group. "This potential collaboration propels forward the development of Everest’s mRNA vaccines, and shows our commitment to bring highly sought-after mRNA vaccines to China."

The lead vaccine candidate for development under Everest’s mRNA technology platform is PTX-COVID19-B, a potentially best-in-class lipid nanoparticle formulated mRNA vaccine with strong immunogenicity and tolerability profiles and has been shown to generate high titer neutralization against the original and variant strains of SARS-CoV-2 in an S protein-typed pseudovirus assays. Based on data from the Phase 1 trial, neutralizing antibody levels at Day 42 were 8.6 times and 23 times higher than convalescence sera in the 40μg and 100μg dose levels, respectively.

Everest’s licensing partner Providence is currently evaluating PTX-COVID19-B in a head-to-head clinical trial against Pfizer-BioNTech’s Comirnaty COVID-19 vaccine. Everest and Providence expect to report top line data in mid-2022, and if positive, this study together with a required safety dataset can support emergency marketing authorization with a stringent Western regulatory authority. Everest and Providence also plan to initiate a registrational booster vaccine trial in 2022 to further expand the indication of PTX-COVID19-B.

About PTX-COVID19-B

PTX-COVID19-B is an mRNA vaccine in Phase 2 development for the treatment of COVID-19, which encodes the full-length S protein of SARS-CoV-2 encapsulated in a lipid nanoparticle (LNP). Interim data from Providence’s Phase 1 study showed that PTX-COVID19-B generated strong virus neutralization activity and produced a level of antibodies in participants in the treatment arm that compare favorably to those produced by other mRNA vaccines that have been approved for use against COVID-19. The treatment was generally safe and well tolerated.

In September 2021, Everest entered into a strategic partnership with Providence Therapeutics Holdings Inc. ("Providence") to advance mRNA vaccines and therapies. Under the terms of the agreement, Everest owns the rights Providence’s mRNA vaccine candidates, including PTX-COVID19-B, in Greater China, Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, Pakistan, Philippines, Singapore, Thailand, Timor-Leste and Vietnam. Everest and Providence also entered into a 50/50 global collaboration under which Everest is enabled to create and develop products using Providence’s mRNA platform for product discovery across a broad range of other prophylactic and therapeutic areas.