On June 23, 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 that it has received approval from the U.S. Food and Drug Administration (FDA) for its ongoing potentially pivotal global study evaluating the efficacy and safety of Berubicin compared with Lomustine (Gleostine) administered after first line therapy for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer (Press release, CNS Pharmaceuticals, JUN 23, 2022, https://www.prnewswire.com/news-releases/cns-pharmaceuticals-receives-approval-from-us-fda-for-protocol-amendment-to-ongoing-potentially-pivotal-global-trial-evaluating-berubicin-for-the-treatment-of-gbm-301573601.html [SID1234616220]).

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"We are pleased to have received this positive response from the FDA and to continue driving the Berubicin clinical development program forward. The key objectives for the protocol amendment are based on the feedback we received from investigators and further consideration of the needs of patients on the study. We have also incorporated the recent WHO classification of glioblastoma1 with pertinent guidelines, ensuring that these patients meet specific criteria that allows us to accurately position the program for success. I am proud of the progress we continue to make on the clinical and regulatory fronts and look forward to further advancing this important trial," commented John Climaco, CEO of CNS Pharmaceuticals.

Berubicin is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier currently being evaluated in a global potentially pivotal study evaluating its efficacy and safety.

The potentially pivotal 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. The primary endpoint of the study is Overall Survival (OS), which is a rigorous endpoint that the 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. Results from the trial will compare Berubicin to a current standard of care (Lomustine), with a 2 to 1 randomization of patients to receive either Berubicin or Lomustine. The amended protocol expands eligibility for the study to patients who have received additional treatments as part of the first line therapy for their disease considering advancements in this area. This change was made due to the complexity of new agents introduced as a component of first line therapy, which allows an additional group of patients that can enroll on the study after what may constitute multiple procedures as their initial treatment.

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

"The operational objectives of this study remain our priority, including using patient reported outcomes, stratification based on MGMT methylation status (a prognostic factor in glioblastomas), and providing both the study drug, Berubicin, as well as the comparator drug, Lomustine, to all sites. With the recent regulatory authority approvals received in Europe, we are currently on the cusp of opening clinical sites globally, including France, Italy, Spain, and Switzerland, to expand the scope and outreach to patients for this trial. We remain dedicated to driving this study forward and ultimately hope to provide a much-needed option for treatment in GBM as a safe and effective therapy," added Sandra L. Silberman, M.D., Ph.D. Chief Medical Officer of CNS Pharmaceuticals.

The FDA has granted CNS Pharmaceuticals Fast Track Designation for Berubicin which enables more frequent interactions with them to provide guidance on expediting the development and review process. Additionally, the Company has 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 June 23, 2022 Bantam Pharmaceutical, a drug discovery and development company targeting selective modulation of mitochondrial dynamics in cancer, reported that new data on the mechanism of action of its lead clinical candidate BTM-3566 will be presented at the Third AACR (Free AACR Whitepaper) International Meeting – Advances in Malignant Lymphoma taking place on the 23-26 June in Boston (Press release, Bantam Pharmaceutical, JUN 23, 2022, View Source;advances-in-malignant-lymphoma-301573728.html [SID1234616219]).

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The studies evaluated the activity of BTM-3566 in tumor lines in vitro and in vivo while further elucidating the mechanism of action.

A PDF of the poster will be available on the Bantam website following the conference.

Michael Stocum, CEO of Bantam Pharmaceutical, commented: "Our data demonstrate exceptional single-agent activity in patient derived xenograft models of difficult-to-treat lymphomas. We are excited to bring this novel mechanism of action to the clinic in the coming months and explore its value as a treatment for relapsed and refractory DLBCL."

Title: Pharmacological activation of the mitochondrial protease OMA1 reveals a therapeutic liability in Diffuse Large B-Cell Lymphoma

About BTM-3566
BTM-3566 is an orally-available novel small molecule compound with broad anti-cancer activity in hematologic and solid tumors, initially focused on Diffuse Large B-cell Lymphomas (DLBCL). BTM-3566’s anti-cancer mechanism of action is unique and differentiated from other therapeutics, disrupting mitochondrial function in tumor cells to induce apoptosis (cell death). An IND application for BTM-3566 in B-cell malignancies is being completed with plans to initiate First in Human trials in lymphoma patients in the fall of 2022.

On June 23, 2022 Berry Oncology, a global leading company specialized in genomic testing and early cancer screening, reported the launch of its innovative one-time precision product HIFI Pan-Cancer Screening, which is an early multi-cancer screening product developed based on the company’s proprietary HIFI technology platform (Press release, Berry Oncology, JUN 23, 2022, View Source [SID1234616218]). With one single testing, the product can accurately detect 6 high-risk and high-incident cancers in China that include lung cancer, esophageal cancer, gastric cancer, liver cancer, pancreatic cancer, and colorectal cancer, with a global leading performance of 87.58% sensitivity, 99.09% specificity and 82% traceability accuracy.

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Cancer has become a global public health challenge. According to the 2020 global cancer burden data released by the International Agency for Research on Cancer (IARC), there were 19.29 million new cancer cases and 9.96 million cancer deaths worldwide in 2020, while the numbers in China were 4.57 million and 3 million respectively in the same year, ranking the first in the world which has imposed a heavy disease burden on individuals and society.

The Healthy China 2030 Plan, issued by the Central Committee of the Communist Party of China and the State Council, clearly states that early diagnosis and treatment of major high-risk cancers is highly encouraged, opportunistic cancer screening should be promoted, and the 5-year overall cancer survival rate in China is expected to be increased by 15% by 2030. In the recently released 14th Five-Year Plan for Bioeconomic Development, it also stated that it is necessary to incorporate advanced technologies such as genetic testing into disease prevention, and to carry out early screening for major diseases such as cancers, so as to provide precision solutions and decision support for individualized treatment.

NGS-based liquid biopsy technology has greatly improved the possibility of early cancer screening. On top of significant improvement in the overall testing performance, innovative liquid biopsy products for early cancer screening, taking HIFI Pan-Cancer Screening as an example, have accessible benefits of minimal invasiveness and user friendliness, which help increase the compliance of high-risk groups and create bigger clinical intervention window for patients with early-stage cancer to improve their survival rate, compared with traditional cancer screening methods such as tumor markers and imaging.

HIFI Pan-Cancer Screening is the first early screening product for multiple cancer types using whole genome sequencing (WGS) of cell-free DNA (cfDNA) worldwide. Compared with other technologies, this technology can capture early cancer signals in a wider range and reduce missed detection caused by individual differences and differences in molecular biological characteristics across various tumor types.

In addition, HIFI Pan-Cancer Screening is developed based on Berry Oncology’s proprietary HIFI technology platform. The self-iterative HIFI technology platform can reduce the costs of product development and testing services, and thus making early screening for multiple cancers more affordable.

Since its establishment, Berry Oncology has been committed to the development of innovative liquid biopsy products for cancer based on the NGS platform. Today, the company has carried out product development covering the full disease course, from early screening and early diagnosis, companion diagnosis, minimal residual disease, to recurrence monitoring.

Zhang Kai, deputy director of the National Cancer Center and the Cancer Prevention Department of the Cancer Hospital of the Chinese Academy of Medical Sciences, said, "With the innovative liquid biopsy technology, we can detect a variety of cancers at one time, making early diagnosis and early treatment more possible for patients, while providing new solutions for China to achieve the goals of cancer prevention and control. However, in addition to technology, the general public still needs to improve their awareness in self screening so that this new technology can be more widely used to truly increase early diagnosis rate and reduce mortality rate."

Oscar Zhang, Principal of Qiming Venture Partners, said, "Qiming has been keeping close company to Berry Oncology since day 1, and has witnessed the upgrades of its HIFI technology platform and its determination in building the cancer prevention and treatment ecosystem. Early cancer screening is a high barrier industry, which examines both technical and execution capabilities of a team from the selection of technical route, the translation of theory to practice, the overall layout of the experiment and so on. The launch of the multi-cancer early screening product will further reinforce Berry Oncology’s leadership in the early screening sector."

Zhou Jun, CEO of Berry Oncology, said, "Since its establishment, Berry Oncology has been dedicated to developing technologies and delivering products for early cancer screening based on the spectrum of malignant tumors, to help more high-risk people identify hidden risks and help more patients prolong survival time and improve quality of life. Moving forward, Berry Oncology will continue to optimize its technology system and iterate early screening products to deliver more advanced early cancer screening solutions in accordance with the actual market demand and environment. In the meanwhile, we will further our partnerships with industry players, universities and research organizations to co-build an ecosystem for cancer prevention and treatment that enables innovations more accessible."

On June 23, 2022 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a biotech company focused on research and development of therapies for difficult-to-treat hematological diseases, reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), has unanimously adopted a positive opinion recommending a full marketing authorization approval (MAA) of Pepaxti (melphalan flufenamide, also called melflufen) in EU (Press release, Oncopeptides, JUN 23, 2022, View Source [SID1234616217]). The European Commission (EC) will make a legally binding decision based on the EMA recommendation within 60 days. Once granted by EC, the marketing authorization is valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Lichtenstein, and Norway.

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The positive opinion is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study which was utilized as confirmatory study. No specific post-marketing commitments were issued. Oncopeptides intends to submit a type II variation in Q4 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).

Pepaxti is indicated, in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

"Pepaxti helps patients with multiple myeloma, an incurable hematologic cancer. Today’s positive CHMP opinion confirms that Pepaxti provides benefit to these patients and is foundational for the future of Oncopeptides and our development pipeline," says Jakob Lindberg, CEO of Oncopeptides. "Based on the scientific evaluation by EMA, our dialogue with the US Food and Drug Administration (FDA) has now been intensified to achieve a clear path forward also for US patients."

Efficacy results for triple-class refractory patients who have received at least 3 prior lines of therapies and who had no ASCT or progressed more than 36 months after an ASCT in the HORIZON study

"The recommendation for full approval of Pepaxti by EMA is really good news for patients with triple class refractory disease, where the unmet medical need remains high and treatment options often are exhausted," says Pieter Sonneveld, professor of Hematology at the Erasmus University Medical Center in Rotterdam, the Netherlands and principal investigator of the OCEAN study.

"EMA´s assessment of Pepaxti corroborates our scientific conclusion that the overall survival result in the OCEAN study constitutes a case of true survival heterogeneity which is reflected in the indication statement in accordance with the agency´s guidelines," says Klaas Bakker, MD, PhD, Executive Vice President, and Chief Medical Officer. "In addition, EMA confirms that there are no toxicological safety signals in both studies and there is a positive benefit risk profile in the indicated patient population. The non-transplanted, often older patient population, which represents the largest group of RRMM patients, particularly benefits from treatment with Pepaxti."

As previously disclosed, Oncopeptides has an EIB loan facility. Oncopeptides and EIB are currently in negotiations, to update tranche definitions to reflect the current regulatory situation. In addition, the Company is considering additional financing options to capture the opportunities with the upcoming EU-approval. This may include new share issues and other public or private financing options.

Oncopeptides will advance market access activities after an approval by the European Commission, to pave the way for a successful launch of Pepaxti in Germany in Q4, 2022. The Company is actively considering various options to commercialize the drug, making it available for patients across Europe, and maximizing shareholder value.

Conference call for investors, analysts, and media 

Investors, financial analysts, and media are invited to participate in a webcast with a Q&A session on June 27, 2022, at 11:00 (CET). The event will be hosted by CEO Jakob Lindberg, CMO Klaas Bakker and CFO Annika Muskantor.

Webcast

The webcast will be streamed via View Source
The link can also be found on the website: www.oncopeptides.com.

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person above, on June 23, 2022, at 17:55 (CET).

About Pepaxti

Pepaxti (melphalan flufenamide, also called melflufen) is a lipophilic peptide conjugated alkylating drug that rapidly and selectively is delivering cytotoxic agents into tumor cells. The drug is composed of a di-peptide and an alkylating moiety. The lipophilicity allows a faster cellular uptake whereas the peptide hydrolysis mediated by aminopeptidases, results in accumulation of alkylating moieties in cancer cells. This results in an improved efficacy without an increased toxicity compared to melphalan. Pepaxti inhibits proliferation and induces apoptosis of haematopoietic and solid tumour cells. It shows synergistic cytotoxicity in combination with dexamethasone in melphalan resistant and non-resistant multiple myeloma cell lines.

Pepaxti is indicated in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapies. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

About Multiple Myeloma

Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cells which produce antibodies to help fight infection, and cause cancer cells to accumulate in the bone marrow. Multiple Myeloma is the second most common hematologic malignancy, and accounts for approximately 1-2% of all new cancer cases, with a global incidence rate of 1.7 per 100,000 and an age-standardized incidence rate of 2.1-3.4 per 100,000 in France, Germany, Italy, Spain, and the UK. An estimated 35,842 patients were diagnosed in the EU27 during 2020, with an estimated 23,275 deaths due to the disease (ECIS 2020).

Patients with multiple myeloma may have symptom-free periods, but the disease always relapses, and patients may become refractory to all available treatment options due to mutations and/or clonal evolution of the tumor cells. A growing subset of patients are triple-class refractory, and develop disease refractory to immunomodulatory drugs, proteasome inhibitors, and CD38- targeting monoclonal antibodies. These patients have a very short expected overall survival.

On June 23, 2022 Paige, a global leader in clinical AI applications in pathology, reported it received CE-IVD and UKCA marks for HER2Complete, an artificial intelligence (AI) software designed to identify patients with breast cancer whose tumors have expressions of human epidermal growth factor receptor 2 (HER2) protein (Press release, Paige AI, JUN 23, 2022, View Source [SID1234616216]).* This is the first CE-IVD and UKCA designated tool to explore the novel space of HER2-low. In a recent study, HER2Complete was able to detect levels of HER2 expression in HER2-negative (IHC-0) and HER2-low (IHC1+/2+) hematoxylin and eosin (H&E)-stained tissue samples, the first and only AI biomarker assay capable of doing so.

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HER2 is a protein that promotes breast cancer cell growth, and breast cancer cells with excess levels of HER2 are called HER2-positive.1 Targeted therapies for HER2-positive tumors have been a mainstay of cancer treatment over the past two decades. Recent evidence suggests there may be a subgroup of patients who are considered HER2-negative through standard immunohistochemistry (IHC) and fluorescence in situ hybridization (FISH) testing but may actually have low or ultra-low levels of HER2, and therefore may be responsive to particular investigative therapies.2,3 It has been challenging to identify these patients reliably and reproducibly using conventional methods, calling for the development of novel ways to assess a patient’s HER2 status that are more accurate and sensitive than traditional assays, which were designed and optimized to detect high levels of HER2 expression.

In contrast to traditional IHC tests for HER2, Paige deploys HER2Complete to detect HER2 expression based on protein and mRNA levels on digital images of H&E-stained tissue samples, with results generated rapidly at the click of a button. Recent work has shown that HER2Complete can also identify HER2 expression in patients currently classified as IHC negative (or IHC-0), in addition to expression in HER2-low (IHC1+ and 2+/FISH negative) patients. This approach complements existing IHC testing to potentially identify true HER2-expressing breast cancers without the need for special staining approaches and with a very rapid turnaround using only the diagnostic biopsy or resection slides. Further, this tool has been used to detect true HER2-negative disease, whose definitive identification based on a lack of IHC and mRNA HER2 expression may accelerate efforts for developing new therapies for this important group of patients who may not benefit from HER2 targeted therapies. This approach doesn’t require any special labelling of the tissue sample and can be done rapidly and easily on standard tissue preparations using methods that already exist in labs across the globe.

"AI brings a transformational approach to diagnostics and allows us to identify low levels of HER2 in tissue that we would not be able to detect using current assays," said David Klimstra, M.D., Founder and Chief Medical Officer at Paige. "We are working to enable the next generation of HER2 testing to provide the information physicians need to guide the use of next generation HER2 therapy. We believe that this assay will allow us to reliably identify increased likelihood of HER2 expression, even on samples where HER2 expression was misclassified by legacy diagnostics as low or null."

"Our product is potentially a very targeted and cost-effective solution to identify a subset of patients who have previously been unidentified," said Jill Stefanelli, Ph.D., President and Chief Business Officer at Paige. "Paige is developing an end-to-end solution to detect and further characterize breast cancer so that pathologists and oncologists get comprehensive insights into an individual’s cancer. We look forward to assessing the clinical utility with multiple potential partners in order to link this test to treatment outcomes."

*In the United States, the software is available for Research Use Only and not for use in diagnostic procedures.