On April 28, 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 Spanish Agency for Medicines and Health Products (AEMPS) Competent Authority and from the CElm Provincial de Sevilla Ethics Committee in Spain 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 28, 2022, View Source [SID1234613189]).

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"We continue to build momentum in our potentially pivotal study of Berubicin, and importantly advance toward bringing a much needed treatment option to patients. We have significantly bolstered our international presence and we are grateful to Spain for joining us in support of this trial. We are committed to driving this development program forward and are executing on all fronts to build momentum," commented John Climaco, CEO of CNS Pharmaceuticals.

Dr. Juan M Sepúlveda Sánchez, MD, 12 de Octubre University Hospital, Madrid, added, "There remains a significant unmet medical need for patients with GBM. We are pleased to bring this important clinical program to Spain and help to further evaluate Berubicin’s potential to meet that need. I look forward to working alongside the rest of the clinical team to advance CNS Pharmaceuticals’ potentially pivotal study and importantly, progress Berubicin through the clinic with the hope of bringing a valuable treatment options to patients and physicians."

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 28, 2022 NEC reported (Press release, NEC, APR 28, 2022, View Source [SID1234613084])

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1. Consolidated Financial Results for the Year ended March 31, 2022 (April 1, 2021 – March 31, 2022)
(1) Consolidated Operating Results
(2) Consolidated Financial Position
(3) Consolidated Cash Flows

2. Dividends
3. Consolidated Financial Results Forecast for the Fiscal Year Ending March 31, 2023 (April 1, 2022 – March 31, 2023)

1. Non-consolidated Financial Results for the Year Ended March 31, 2022 (April 1, 2021 – March 31, 2022)
(1) Non-consolidated Operating Results
(2) Non-consolidated Financial Position

*This consolidated financial results falls outside the scope of audit to be performed by certified public accountants or an audit firm.

*Explanation concerning the appropriate use of the financial results forecast and other special matters (Adjusted profit (loss)) "Adjusted operating profit (loss)" is an indicator for measuring underlying profitability in order to clarify the contribution of acquired companies to the NEC Group’s overall earnings. It is measured by deducting amortization of intangible assets recognized as a result of M&A and expenses for acquisition of companies (financial advisory fees and other fees) from operating profit (loss). Also, "Adjusted net profit (loss) attributable to owners of the parent" is an indicator for measuring underlying profitability attributable to owners of the parent. It is measured by deducting adjustment items of operating profit (loss) and corresponding amounts of tax and non-controlling interests from net profit (loss) attributable to owners of the parent. (Cautionary statement with respect to forward-looking statements) The forward-looking statements such as operating results forecast contained in this statements summary are based on the information currently available to NEC Corporation ("the Company") and certain assumptions considered reasonable. Actual operating results may differ significantly from these forecasts due to various factors.

For details, please refer to "3. Cautionary Statement with Respect to Forward-Looking Statements" on page 16. (How to obtain supplementary financial materials and information on the financial results briefing) On April 28, 2022, the Company will hold a financial results briefing for the institutional investors and analysts. Presentation materials will be posted on the company website after the release of financial results, and the presentation video and Q&A summary will be also posted on the company website promptly after the financial results briefing. In addition to the above, the Company periodically holds briefings on business and operating results for the individual investors. Presentation materials and Q&A summary will be posted on the company website promptly after the briefing. For the schedule and details, please check the company website.

On April 28, 2022 XBiotech (NASDAQ: XBIT) reported that the French National Agency for the Safety of Medicines and Health Products [L’Agence nationale de sécurité du médicament et des produits de santé (ANSM)] approved the launch of a multicenter randomized clinical study for XBiotech’s candidate cancer treatment Natrunix in combination with trifluridine/tipiracil for the treatment of colorectal cancer (Press release, XBiotech, APR 28, 2022, View Source [SID1234613124]). The French National Cancer Institute (INCA) has also awarded a grant to fund all clinical costs for the study.

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Investigators will combine Natrunix and trifluridine/tipiracil as a new candidate therapy for metastatic colorectal cancer in subjects that have failed earlier treatment with oxaliplatin, irinotecan, and fluoropyrimidine. The study will randomize patients to receive the Natrunix plus chemotherapy or placebo plus chemotherapy and is designed to seamlessly proceed to a Phase III study based on achievement of certain early efficacy milestones.

Headed by Dr. François Ghiringhelli and Dr. Come Lepage, the clinical program will include over 20 participating clinical centers and enroll at least 160 subjects. Dr. François Ghiringhelli is Professor in Medical Oncology and Director of the INSERM research team at the Georges-Francois Leclerc Cancer Centre, and Dr. Come Lepage, Prof. Department Gastroenterology and Digestive Oncology, University Hospital Dijon, Dijon, France. The study design was developed by the lead investigators in collaboration with XBiotech.

The first portion of the study will be an open label, dose escalation (3:3). The Phase II portion will be a multicenter, randomized, double blind, placebo-controlled, non-comparative trial that will enroll 160 subjects. The main objective of the phase II study is to evaluate the efficacy of Natrunix + trifluridine/tipiracil in comparison with placebo + trifluridine/tipiracil with respect to 6-month overall survival in patients with refractory metastatic colorectal cancer. Secondary efficacy measures in the phase 2 portion will include progression free survival, median overall survival, tolerance, quality of life, serum markers of inflammatory cytokines, and tumor markers by immunohistochemistry. With successful completion of the primary endpoint in the Phase II portion, the study will continue into a phase III trial with the number of additional patients enrolled based upon results from the Phase II.

Natrunix is a therapeutic monoclonal antibody discovered, manufactured and undergoing clinical development by XBiotech. The antibody blocks the activity of interleukin-1 alpha (IL-1α). Malignant tumors "trick" the body into producing IL-1α, which has multiple roles in supporting tumor growth and spread. IL-1α expression is also induced by cytotoxic chemotherapy. IL-1α is a potent activator of new blood vessel formation (upregulating VEGF and tumor neoangiogenesis); it mediates breakdown of connective tissue through stimulating matrix metalloproteinase production; facilitates metastasis (enhancing adhesion and migration across blood vessels); and mediates systemic illness (fatigue, anorexia, and anxiety) through activating the hypothalamic-pituitary-adrenal axis. Using Natrunix to block IL-1α in combination therapy could inhibit tumor growth and spread, reduce unwanted effects of chemotherapy, and improve outcomes.

Colorectal cancer is one of the most common forms of cancer in Europe and the United States, with the American Cancer Society’s estimating over 151,000 new cases and over 52,000 deaths in the United States alone.

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

On April 28, 2022 Tvardi Therapeutics, Inc. ("Tvardi"), a privately held, clinical-stage biopharmaceutical company focused on the development of STAT3 inhibitors, reported that management will participate in one-on-one meetings with investors at the Morgan Stanley Private Company Biotech Corporate Access Day (Press release, Tvardi Therapeutics, APR 28, 2022, View Source [SID1234613141]). The conference is being held in a virtual format on Tuesday, May 3rd, 2022.

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On April 28, 2022 The Janssen Pharmaceutical Companies of Johnson & Johnson reported the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of niraparib in combination with abiraterone acetate, in the form of a dual action tablet (DAT)* plus prednisolone, for the treatment of patients with prostate cancer who have progressed to metastatic castration-resistant prostate cancer (mCRPC) and are positive for homologous recombination repair (HRR)+ gene alterations (Press release, Johnson & Johnson, APR 28, 2022, View Source [SID1234613174]). When approved by the European Commission, niraparib in combination with AAP will be the first dual action tablet formulation in the European Union specifically targeting HRR gene alterations in mCRPC.

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The combination of niraparib, a PARP (poly adenosine diphosphate-ribose polymerase) inhibitor, and abiraterone acetate, a CYP17 inhibitor, targets two oncogenic drivers in patients with mCRPC, AR-axis and HRR gene alterations. The DAT formulation is also intended to be more convenient for patients, and thus aims to improve treatment compliance. Prostate cancer is one of the most common cancers in Europe with approximately 473,000 patients diagnosed in 2020.2 Up to approximately 30% of patients with mCRPC have HRR gene alterations which are associated with a worse prognosis compared to patients without HRR gene alterations.1

"People with prostate cancer harbouring BRCA alterations face a more aggressive form of disease with worse outcomes and faster progression, sadly leading to a shorter life expectancy," commented Professor Gerhardt Attard=, Primary Study Investigator and Clinician Scientist and Team Leader at University College London Cancer Institute. "This submission is an important step towards improving the outcomes for people with metastatic prostate cancer harbouring BRCA alterations using a targeted therapy that significantly delays the time to their cancer progressing."

The EU MAA is supported by data from the MAGNITUDE study (NCT03748641), a Phase 3, randomised, double-blind, placebo-controlled, multicentre study evaluating the safety and efficacy of niraparib combined with abiraterone acetate plus prednisone (AAP) in patients with mCRPC. The study showed that at the final analysis for radiographic progression-free survival (rPFS), the treatment combination of niraparib and AAP demonstrated a statistically significant improvement in patients with HRR gene alterations as compared to placebo and AAP.1 First results from the study were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) – Genitourinary Cancers Symposium (ASCO GU 2022) Annual Meeting (Abstract #12). The study continues to collect data on the secondary endpoints, which include time-to-initiation of cytotoxic chemotherapy, time to symptomatic progression and overall survival.1

"The data supporting this submission demonstrate the benefit of niraparib in combination with AAP in patients with specific gene alterations and reinforce the importance of biomarker testing in helping to provide an individualised treatment for these patients," said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. "We are committed to advancing targeted therapeutic options for patients with prostate cancer as we build upon our deep understanding of the disease, with a focus on improving outcomes for patients."

"The submission of niraparib in combination with AAP to the European Medicines Agency marks an important milestone in addressing specific genetic alterations in prostate cancer," said Mathai Mammen, M.D., Ph.D., Executive Vice President, Pharmaceuticals, R&D, Johnson & Johnson. "We are determined to transform this complex disease through innovation, science and ingenuity."

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About Niraparib
Niraparib is an orally administered, selective poly-ADP ribose polymerase (PARP) inhibitor, that is currently being studied by Janssen for the treatment of patients with prostate cancer.1 Additional ongoing studies include the Phase 3 AMPLITUDE study evaluating the combination of niraparib and AAP in a biomarker-selected patient population with metastatic hormone-sensitive prostate cancer (mHSPC).3

In April 2016, Janssen Biotech, Inc. entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc. (acquired by GSK in 2018), for exclusive rights to niraparib in prostate cancer. In the European Union, niraparib is indicated for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to first-line platinum-based chemotherapy; for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy (Zejula SmPC 2021). Niraparib is currently marketed by GSK as ZEJULA.4

About abiraterone acetate
Abiraterone acetate is an orally-administered androgen biosynthesis inhibitor. In the European Union, abiraterone acetate is indicated with prednisone or prednisolone for the treatment of newly diagnosed high risk mHSPC in adult men in combination with ADT; the treatment of mCRPC in adult men who are asymptomatic or mildly symptomatic after failure of ADT in whom chemotherapy is not yet clinically indicated; and the treatment of mCRPC in adult men whose disease has progressed on or after a docetaxel based chemotherapy regimen (ZYTIGA SmPC 2020).5

Abiraterone acetate is currently marketed by Janssen Janssen-Cilag International NV as ZYTIGA.5

About Metastatic Castration-Resistant Prostate Cancer
Metastatic castration-resistant prostate cancer (mCRPC) characterises cancer that no longer responds to ADT and has spread to other parts of the body. The most common metastatic sites are bones, followed by lungs and liver.6 Prostate cancer is the most common cancer in men in Europe.7 More than one million men around the world are diagnosed with prostate cancer each year.8 Patients with mCRPC and HRR gene alterations have a worse prognosis than those without HRR alterations.9

About MAGNITUDE
MAGNITUDE (NCT03748641) is a Phase 3 randomised, double-blind, placebo-controlled, multicentre clinical study evaluating the safety and efficacy of the combination of niraparib and AAP for patients with mCRPC, with or without certain HRR gene alterations. The study includes two cohorts in which patients were randomised to receive either niraparib and AAP or placebo and AAP cohorts: one cohort of patients with predefined HRR gene alterations (including ATM, BRCA1, BRCA2, BRIP1, CDK12, CHEK2, FANCA, HDAC2, PALB2 alterations) and one cohort of patients without HRR gene alterations. In a third, open-label cohort, all patients received the dual action tablet formulation of niraparib and AAP.1

The primary endpoint of the MAGNITUDE trial is rPFS. Secondary endpoints include time-to-initiation of cytotoxic chemotherapy, time to symptomatic progression and overall survival.1