On September 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 the activation of its first clinical trial sites in Europe for the ongoing potentially pivotal global trial evaluating Berubicin for the treatment of recurrent glioblastoma multiforme (GBM), one of the most aggressive types of brain cancer (Press release, CNS Pharmaceuticals, SEP 28, 2022, View Source [SID1234621532]). These first European clinical trial sites in France and Spain are now open and actively enrolling patients.

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"Our primary focus continues to be on advancing the clinical development of Berubicin as a potential treatment option for this devastating disease. Over the course of this year, we have worked diligently to expand patient eligibility for our potentially pivotal trial and bolster our international presence now realized with the activation of the first two European clinical trial sites. This is a true testament to our team’s commitment to and the execution of this important clinical program. We are grateful for the tremendous support from the clinical staff and remain dedicated to driving patient enrollment as quickly and efficiently as possible. We look forward to continuing to build momentum and bringing Berubicin across the finish line to unlock its greatest potential. As long as the unmet medical need in GBM remains, we will continue our fight in earnest to bring hope to patients and families globally," commented John Climaco, CEO of CNS Pharmaceuticals.

Berubicin, is a novel anthracycline and the first anthracycline to appear to cross the blood-brain barrier. Berubicin is currently being evaluated in a potentially pivotal global study evaluating its efficacy and safety in the treatment of GBM. The potentially pivotal trial is an adaptive, multicenter, open-label, randomized and controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV1) 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 recently 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 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 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.

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 September 28, 2022 CNBX Pharmaceuticals Inc. ( OTCQB: CNBX), a global leader in the development of cancer related cannabinoid-based medicine, reported that it has received Notice of Grant for Patent by IP Australia, the Australian Government (Press release, CNBX Pharmaceuticals, SEP 28, 2022, View Source [SID1234621531]). Said notice relates to Company’s patent titled: System and Method for High Throughput Screening of Cancer Cells. The term of the patent granted is set for 20 years starting May 2016 and until May of 2036.

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This patent now becomes part of the company’s growing IP portfolio, including 8 patent families, with 7 granted patents and 18 additional pending. The company’s IP portfolio is focused on cancer patients and cancer treatments, and includes claims relating to Pharmaceutical Compositions, Methods and Systems.

On September 28, 2022 Verismo Therapeutics, a clinical-stage CAR-T company and Penn spinout, and pioneer of the novel KIR-CAR platform technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SynKIR-110 for the treatment of patients with mesothelin-expressing mesotheliomas (Press release, Verismo Therapeutics, SEP 28, 2022, View Source [SID1234621530]).

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SynKIR-110 is a next generation approach to cell therapy targeting solid tumors, and the first product to use the novel KIR-CAR platform, a modified NK-like receptor designed to improve persistence and efficacy against aggressive solid tumors. Phase 1 clinical trials of SynKIR-110 in patients with mesothelin-expressing ovarian cancer, mesothelioma and cholangiocarcinoma will commence in Q1 2023.

"Orphan Drug Designation is another major milestone for Verismo following the acceptance of our SynKIR-110 IND," said Dr. Bryan Kim, CEO of Verismo. "Mesothelioma is a rare and deadly disease for which few other treatment options currently exist. We look forward to working with the mesothelioma community to advance SynKIR-110 as a potential treatment while we continue to expand clinical investigation of this novel platform in other cancers in the solid tumor space."

Orphan Drug Designation is a status given to drugs intended to treat a rare disease that affects fewer than 200,000 people in the United States and show promise in the treatment, prevention, or diagnosis of that disease. Benefits to the study sponsor include seven years of marketing exclusivity, federal tax credits, waiver of Prescription Drug User Fee Act (PDUFA) fees and increased regulatory assistance from the FDA.

The Phase 1 trial for SynKIR-110, STAR-101 (SynKIR T cell Advanced Research), will begin enrolling patients in the first quarter of 2023 at the initial clinical site, the Hospital of the University of Pennsylvania.

About the KIR-CAR Platform
The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical models to be capable of maintaining antitumor T cell activity even in challenging solid tumor environments. DAP12 costimulatory chains aid additional T cell stimulating pathways, further improving cell persistence. This continued function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those refractory to traditional CAR T cell therapies. Furthermore, the KIR-CAR platform can be combined with many additional emerging technologies, such as in vivo gene editing, advanced T cell selection and modulation, combination therapies, and even allogeneic cellular therapies to provide an adaptable targeted anti-tumor therapy for patients in need.

About Mesothelioma
Mesothelioma is a rare type of cancer that affects the lining of the chest or abdominal cavities. More than 2,000 cases of this aggressive cancer are diagnosed in the U.S. each year. While life expectancy can differ depending on a variety of factors, such as type and location of the disease, stage at diagnosis, and general patient health, overall, patients usually survive only four to eighteen months after diagnosis.

On September 28, 2022 Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel small molecule therapies that address emerging targets in oncology, reported financial results for the first half of 2022 and provided a corporate update (Press release, Ryvu Therapeutics, SEP 28, 2022, View Source [SID1234621529]).

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In August, Ryvu secured €22 million in funding from the European Investment Bank to support Ryvu’s pipeline development.
In July, Ryvu signed an exclusive license agreement with Nasdaq-listed Exelixis to develop novel STING agonist-based targeted cancer therapies.
RVU120 clinical and preclinical data were presented at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress.
In August, Ryvu presented its development plans, including broad development for RVU120 in hematologic and solid tumors and preclinical advancement of the synthetic lethality and immuno-oncology pipeline.
On September 19, Ryvu’s shareholders voted in favor of a resolution granting the Management and Supervisory Board the right to issue authorized capital, providing an important potential source of future financing.
– The first half of 2022 has been productive and progressive for Ryvu. Investors have appreciated the progress in our research and partnering and have supported our proposal to issue authorized capital at our discretion to finance development plans over the coming years. We are happy to have a quality investor base with such a great trust and understanding of our needs and potential – said Pawel Przewiezlikowski, co-founder, largest shareholder and CEO of Ryvu Therapeutics.

FIRST HALF 2022 AND RECENT HIGHLIGHTS

– Despite challenging market conditions, our ability to secure additional debt financing and establish an important new corporate partnership reflects the quality of our scientific expertise and clinical development capabilities – said Pawel Przewiezlikowski.

European Investment Bank (EIB) €22 million financing to accelerate the development of blood cancers and solid tumor therapies: The funding is provided under the EIB’s venture debt instrument and will finance Ryvu’s pipeline development of new cancer treatments. The funding is guaranteed by the European Fund for Strategic Investments, the financial pillar of the Investment Plan for Europe.

Exclusive License Agreement with Exelixis to develop new targeted therapies utilizing Ryvu’s STING (STimulator of INterferon Genes) technology: Under the terms of the agreement, Exelixis paid Ryvu an upfront fee of $3 million in exchange for certain rights to Ryvu’s STING agonist small molecules. Exelixis will be responsible for all research, development, and commercialization activities for which Ryvu will be eligible to receive milestone payments, as well as tiered royalties on the annual net sales of any products that are successfully commercialized under the collaboration.

Promotion of Ryvu’s Chief Medical Officer, Hendrik Nogai, and Chief Business Officer, Vatnak Vat-Ho to the Management Board: Dr. Nogai has served as Chief Medical Officer at Ryvu since February 2022 and Mr. Vat-Ho has been Ryvu’s Chief Business Officer since April 2021. The promotions reflect each individual’s significant contributions toward advancing Ryvu’s lead candidate, RVU120, and expanding Company’s business development activities.

Presentation of Clinical and Preclinical Data on RVU120 at the 2022 European Hematology Association (EHA) (Free EHA Whitepaper) Congress: Data from the ongoing Phase 1b dose-escalation study of RVU120 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (HR-MDS) demonstrated single-agent activity with one complete remission (CR) and stable diseases (SD) with blast reductions in several ongoing patients who failed multiple prior lines of therapy.

Additionally, data on SEL24/MEN1703, an orally available dual PIM/FLT3 inhibitor was presented. SEL24/MEN1703 was well tolerated, with no drug discontinuations or deaths due to treatment-related adverse events (TRAEs) in patients with relapsed or refractory (R/R) IDHm AML. Promising efficacy was observed, with overall response rates (ORR) and complete remission (CR) / CR with incomplete hematologic recovery (CRi) / CR with partial hematologic recovery (CRh) of 13% for the IDHm cohort.

Key Opinion Leader Webinar on RVU120 for the Treatment of Hematological Malignancies and Solid Tumors: The webinar featured a presentation by KOL Dr. Michael Savona, MD, from Vanderbilt University School of Medicine, who discussed the current treatment landscape and unmet medical need in treating patients with AML and HR-MDS. Additionally, Ryvu’s leadership team discussed the underlying mechanism of action of RVU120 and provided an update on the Phase I data for RVU120.

Project updates from RVU120 and SEL24 programs presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting: Updates from the Phase 1/2 study of SEL24/MEN1703, a first-in-class dual PIM/FLT3 kinase inhibitor, in patients with IDH1/2-mutated acute myeloid leukemia and Phase I/II trial of RVU120, a CDK8/CDK19 inhibitor in patients with relapsed/refractory metastatic or advanced solid tumors were presented.

Poster Presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting: Data were presented from the RVU120 Trial-In-Progress in patients with relapsed/refractory metastatic or advanced solid tumors and in vitro and in vivo data demonstrating RVU120 efficacy against hormone-independent breast cancer. Additionally, a novel MTA-cooperative PRMT5 inhibitor as a targeted therapeutic for MTAP deleted cancer was presented.

Shareholders meeting convened and approved authorized capital on September 19: At an extraordinary shareholders meeting, shareholders voted in favor of a resolution to approve authorized capital, which provides the Management and Supervisory Board the flexibility to issue equity at its discretion when optimal.

– We are really appreciative of the overwhelming support that we received at our shareholders’ meeting, which further enables Ryvu to execute our mission of discovering and developing drugs that will improve the lives of cancer patients and their families while maximizing the Company’s value – underlined Pawel Przewiezlikowski.

UPCOMING CLINICAL AND CORPORATE MILESTONES:

RVU120

Data update from the ongoing Phase I clinical study in acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS) and the ongoing Phase I clinical study in solid tumors will be presented in Q4 2022
Synthetic lethality

Data on Ryvu’s novel MTA-cooperative PRMT5 inhibitors as targeted therapeutics for MTAP deleted cancers to be presented in Q4 2022.

In August, Ryvu also highlighted its development plans for 2022-2024, which are focused on rapidly advancing its pipeline to treat cancer patients:

Broad development of RVU120, including a potential fast-to-market strategy in AML/HR-MDS,
Advancing into Phase I clinical trials one additional program,
Strengthening of the Synthetic Lethality Platform and discovery of novel targets,
Acceleration in the early pipeline,
Executing milestones from existing collaborations (i.e. Menarini, Galapagos, Exelixis), and adding at least one new partnering deal per year.
FIRST HALF 2022 FINANCIAL UPDATE:

Cash Position – On September 21, 2022, Ryvu Therapeutics held $9.7M in cash, cash equivalents, and short-term investments, compared to $20.5M at the end of 2021.

Operating costs, excluding the non-cash cost of valuation of the Incentive Program ($3.8M) and valuation of NodThera shares ($1.8M), for the 6 months period ended June 30, 2022, amounted to $13M and related primarily to research and development expenditures, while the operating costs without Incentive Program and valuation of Nodthera shares for the same period last year amounted to $11.5M.

Net Loss Attributable to Common Shareholders – Net loss attributable to common shareholders excluding the non-cash cost of valuation of the Incentive Program was $11M for the 6 months period ended June 30, 2022, as compared to a net loss without the Incentive Program of $8.2M for the same period last year.

On September 28, 2022 The Precision Cancer Consortium (PCC) reported the addition of two new members, Lilly and the Janssen Pharmaceutical Companies of Johnson & Johnson (Press release, The Precision Cancer Consortium, SEP 28, 2022, View Source [SID1234621528]). The PCC now consists of six member organizations including founding members Bayer, GSK, Novartis, and Roche who are collaborating to support efforts to improve patient access to comprehensive genomic testing in routine care and in clinical trials globally. The PCC will drive a variety of initiatives aimed at increasing patient access to precision diagnostics using comprehensive genomic testing, including next generation sequencing (NGS).

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Aligned with one of PCC’s key aims to remove barriers to access and increase awareness of the benefit of genomic testing for healthcare stakeholders, the first educational session "Bringing Precision Oncology Closer to Community Care" will be held virtually on September 29th, 2022 at 16:00 CET. Join us for our interactive peer to peer workshop, the event is open to all, register here.