On June 8, 2022 Harbour BioMed ("HBM", HKEX: 02142) reported that China National Medical Products Administration (NMPA) had approved the investigational new drug (IND) application to commence phase I trial of its B7H4x4-1BB bispecific antibody (HBM7008) in China (Press release, Harbour BioMed, JUN 8, 2022, View Source [SID1234615792]). This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary anti-tumor activity of HBM7008 in patients with solid tumors. It has successfully completed the dosing of first patient in the phase I trial of HBM7008 in Australia on 25 May 2022.

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HBM7008 is generated from HBM’s unique and innovative HBICE platform. It is a first-in-class bispecific antibody targeting B7H4 and 4-1BB. The bispecific antibody can engage and activate T cells by 4-1BB only in B7H4 positive tumor microenvironment. B7H4 is overexpressed on a variety of solid malignancies, including breast, ovarian, endometrial, and non-small cell lung cancers. With its crosslinking-dependent specificity on tumors and potent immune modulation activity, HBM7008 has shown excellent safety profile with strong anti-tumor efficacy in the pre-clinical study, including completed response observed in the mouse tumor model.

"4-1BB is one of the most promising anti-tumor immune targets, providing new solutions for cancer treatment. As the first-in-class bispecific antibody targeting B7H4 and 4-1BB, HBM7008 is expected to lead the development of next-generation immunotherapeutics. Based on preclinical study data, we are highly confident in B7H4x4-1BB bispecific antibody. We will efficiently promote this clinical study to provide a novel, effective and safe treatment for patients, so that more cancer patients can benefit from the innovative therapeutic." said Dr. Humphrey Gardner, CMO of Harbour BioMed.

About HBM7008

HBM7008 is a bispecific antibody targeting Tumor-Associated Antigen B7H4x4-1BB that not only displays high potency in the T cell co-stimulation and tumor growth inhibition, and potentially may also translate to better safety due to its strict dependency of TAA-mediated crosslinking T cell activation. HBM7008 is one of the fully human bispecific antibodies developed from the HBICE platform of the Company. It is the only bispecific antibody against these two targets globally. Its unique specificity on tumors and immune modulation activity makes it a promising therapeutic in PD-L1 negative or PD-1/PD-L1 resistant patients. It also has the potential to avoid 4-1BB liver toxicity risk observed in other products with the benefit of its innovative biology mechanisms and bispecific design.

On June 8, 2022 Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, reported the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for TP-3654, the company’s proprietary investigational oral inhibitor of PIM kinases, for the treatment of myelofibrosis (Press release, Sumitomo Pharmaceuticals, JUN 8, 2022, View Source [SID1234615791]).

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"This designation is an important milestone in the development of TP-3654 and highlights the need for potential new treatment options for patients with myelofibrosis," said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. "This rare hematologic cancer can also progress and worsen. We are excited about collaborating with investigators to advance this clinical-stage asset with the goal of improving patient outcomes."

The FDA’s Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Myelofibrosis is a rare type of bone marrow cancer which disrupts an individual’s normal production of blood cells.1 Myelofibrosis has approximately 1.5 reported cases per 100,000 people each year in the United States.2

"TP-3654 is an investigational oral inhibitor of PIM kinases. PIM kinases have potential antitumor and anti-fibrotic effects through multiple pathways, including induction of apoptosis.3,4 Notably, PIM kinase expression correlates with increased cell survival and reduced apoptosis in tumors, supporting the potential of PIM kinases as novel therapeutic targets,"3 explained Jatin J. Shah, M.D, Chief Medical Officer of Sumitomo Pharma Oncology, Inc. "PIM-1 expression is significantly elevated in myelofibrosis hematopoietic cells and therefore a potential therapeutic target for myelofibrosis."4

TP-3654 is currently being evaluated in a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with intermediate or high-risk primary or secondary myelofibrosis. It is being conducted in the United States and Japan. To learn more about the study and eligibility for enrollment visit clinicaltrials.gov (NCT04176198).

About TP-3654
TP-3654 is an oral investigational inhibitor of PIM kinases, which has potential antitumor and anti-fibrotic effects through multiple pathways, including induction of apoptosis.3,4 TP-3654 inhibited proliferation and increased apoptosis in murine and human hematopoietic cells expressing clinically relevant JAK2V617F mutation.4 TP-3654 alone and in combination with ruxolitinib normalized WBC and neutrophil counts, and reduced spleen size and bone marrow fibrosis in JAK2V617F and MPLW515L murine models of myelofibrosis.4 TP-3654 is currently being evaluated in two clinical trials; A Phase 1 study to treat TP-3654 in patients with advanced solid tumors (NCT03715504); A Phase 1/2 study of oral TP-3654 in patients with intermediate and high-risk myelofibrosis (NCT04176198).

On June 8, 2022 Glympse, a biotechnology company developing revolutionary technology to diagnose and monitor disease, reported that it will have two poster presentations demonstrating that its novel protease biosensor diagnostic platform can detect hepatocellular carcinoma (HCC) at the European Association for the Study of the Liver (EASL) International Liver Congress 2022 (ILC) (Press release, Glympse Bio, JUN 8, 2022, View Source [SID1234615790]). The conference will be held from June 22-26 at ExCeL in London, United Kingdom.

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HCC is the fifth leading cancer globally, and non-invasive diagnostic options are needed to improve early diagnosis for better treatment outcomes. Glympse has developed a novel diagnostic platform technology that utilizes biosensors and machine learning to measure protease activity in plasma samples and will present two analyses demonstrating the platform’s ability to detect HCC. In poster #SAT564, Glympse will present results from a study of two independently tested, diverse cohorts of patients (n = 94) with known HCC vs. healthy controls, which determined Glympse’s platform was effective at detecting HCC (AUC ≥ 0.94). In the second poster (#FRI236), Glympse utilized the platform technology to differentiate between plasma samples with diagnosed HCC and without HCC but with cirrhosis (n = 43 and 26, respectively). Results showed that the protease activity platform was effective at HCC detection (AUC = 0.93) and has potential future utility for early diagnosis, considering 72% of the HCC cases had early-stage tumors.

"Our data demonstrate that the Glympse protease-based diagnostic platform is showing very promising data in the early detection of HCC," said Tram Tran, M.D., Chief Medical Officer of Glympse. "With proper validation, this technology could be used in surveillance strategies for earlier, easier, and more accurate diagnosis of HCC."

"The Glympse diagnostic platform continues to demonstrate highly accurate predictive capabilities for disease detection," said Caroline Loew, Ph.D., Chief Executive Officer of Glympse. "Following our presentation at AASLD, in which we were able to predict NASH vs. healthy patients with 97% accuracy, these data at the EASL conference showcase the platform’s ability to highly accurately detect HCC. We look forward to the continued validation of this platform, and of the measurement of protease activity, to accurately diagnose and monitor disease for patients in need."

Information regarding the two poster presentations can be found below.

Title: Novel and accurate measurement of differential protease activity in diagnosed HCC patients compared to non-HCC cirrhotic patients
Presented By: Amit Singal, M.D.
Poster Session: Non-invasive assessment of liver disease except NAFLD
Time/Date: June 24, 2022, 9:00 a.m. – 6:30 p.m. BST
Abstract #: FRI236

Title: Measurement of protease activity using novel plasma biosensors can accurately detect HCC
Presented By: Tram Tran, M.D.
Poster Session: Liver tumours: Clinical aspects except therapy
Time/Date: June 25, 2022, 9:00 a.m. – 6:30 p.m. BST
Abstract #: SAT564

On June 8, 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 Sigmund Hsu, MD, member of the Company’s Scientific Advisory Board, presented an overview of its ongoing clinical trial evaluating Berubicin for the treatment of recurrent glioblastoma multiforme (GBM), at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2022 Annual Meeting (Press release, CNS Pharmaceuticals, JUN 8, 2022, View Source [SID1234615789]).

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The abstract titled "Design and initiation of an adaptive, randomized, controlled study of berubicin, a topoisomerase 2 poison that crosses the blood brain barrier (BBB), for the treatment of recurrent glioblastoma multiforme (GBM) after first-line therapy," was presented in the Central Nervous System Tumors poster session by Dr. Hsu.

Berubicin is a novel anthracycline and the first that appears to cross the blood-brain barrier (BBB) with central nervous system (CNS) uptake. The induction of apoptosis and DNA damage by Berubicin was significantly higher than that of doxorubicin (Dox) in all tested cancer cells, demonstrating Berubicin’s greater potential potency and consistently higher cytotoxicity in GBM cell lines than Dox. In models of intracranial orthotopic gliomas, Berubicin prolongs survival when compared to temozolomide (TMZ), currently the standard of care in GBM for first line therapy in combination with radiation. Further evaluation of the orthotopic glioma models demonstrated that Berubicin had greater infiltration into intracranial glioblastoma cells compared to normal tissue, supporting the potential for improved efficacy. TMZ has been shown to be less effective in approximately 50% of patients due to changes (methylation) to a DNA repair enzyme, MGMT, that when unmethylated proves to be a prognostic indicator of a poorer prognosis. These differences have been proven to not be a factor in the activity of Berubicin.

Based on this data, a Phase 1 dose escalation study was conducted by a prior developer in patients with recurrent primary brain tumors. Berubicin was well tolerated, with myelosuppression (neutropenia and thrombocytopenia) as dose-limiting toxicities. Of 25 patients evaluable for efficacy, there was 1 complete response (14+ years), 1 partial response durable for 12 weeks, and 9 patients with stable disease over 6 weeks for a clinical benefit rate of 44%.

"We are fully dedicated to advancing the development of Berubicin toward potentially providing an innovative and much-needed option for treatment in GBM, an area of significant unmet medical need. With this goal in mind, we consider that our global study, which has been thoughtfully designed based on prior data as well as input from key opinion leaders and feedback from the FDA, could potentially be pivotal. We also believe that valuable interactions with our investigators on this study, as well as our heartfelt consideration of the needs of patients has positioned us for a successful outcome," added Dr. Sandra L. Silberman, M.D., Ph.D., Chief Medical Officer of CNS Pharmaceuticals. "Moreover, the positive feedback we’ve received from the regulatory authorities across Europe for this trial bolsters confidence in our state-of-the-art trial design and will significantly expand our ability to reach these patients."

The Company’s ongoing global study is an adaptive, multicenter, open-label, randomized, controlled study in adult patients with recurrent glioblastoma multiforme (WHO Grade IV) after failure of standard first-line therapy evaluating efficacy and safety of Berubicin. The primary endpoint of this study, being conducted in the United States and Europe, is overall survival (OS), with a projected 243 patients enrolled in a 2:1 randomization design (Berubicin:Lomustine). This study has pharmacokinetic (PK) evaluations of all patients enrolled, with 15 patients undergoing complete PK assessments throughout the dosing period. Patients will be stratified on the basis of MGMT methylation, documentation of IDH mutational status that now defines Grade IV GBM. No prior administration of bevacizumab will be allowed. An interim analysis will evaluate the comparative effectiveness of these treatments, an adaptive design intended to demonstrate that Berubicin’s efficacy is at least equal to that of Lomustine such that continuation of the study is in patients’ best interests (futility analysis). The overall survival endpoint and sample size have been calculated to be able to show a statistical difference between the two therapies as second line treatment for GBM. Additional studies in malignant diseases of the CNS (e.g., pediatric brain tumors, primary CNS lymphoma, metastatic tumors) are also being explored based on the potential for anthracycline activity in these indications.

For more information about this 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 8, 2022 Alpha Tau Medical Ltd. (Nasdaq: DRTS and DRTSW), ("Alpha Tau" or the "Company"), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT, reported that the U.S. Food and Drug Administration (FDA) has conditionally approved the Company’s Investigational Device Exemption (IDE) application to initiate its multi-center pivotal study for the treatment of recurrent cutaneous Squamous Cell Carcinoma (SCC) using the Alpha DaRT (Press release, Alpha Tau Medical, JUN 8, 2022, View Source [SID1234615788]).

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The clinical study, entitled "A Prospective Multicenter, Pivotal, Single Arm, Open Label Clinical Study to Assess the Efficacy and Safety of Intratumoral Alpha DaRT for the Treatment of Patients with Recurrent Cutaneous Squamous Cell Carcinoma" has been approved to enroll up to 86 U.S. patients at up to 20 institutions in the U.S., in addition to any sites that may be added outside the U.S., and will focus on patients with recurrent cutaneous SCC who have failed at least first line standard of care therapy and are not indicated for another curative standard of care therapy.

"We are very excited about the upcoming commencement of our U.S. pivotal study, undoubtedly our most significant clinical trial to date," commented Alpha Tau CEO Uzi Sofer. "Our receipt of FDA conditional approval of our IDE submission is a critical milestone on our journey to bringing this exciting new therapy to patients in the U.S. and around the world"

Dr. Robert B. Den, Alpha Tau Chief Medical Officer, added, "We are thrilled to have received FDA conditional approval of our IDE, after tremendous focus on the U.S. over the past year, including successful completion of our pilot study and the upcoming initiation of this pivotal study under the FDA’s Breakthrough Device Designation Program. We have seen tremendous engagement and excitement from dozens of leading institutions in the U.S. and around the world, keen to be part of this study, and look forward to working with them on this clinical study."

Yaniv Sagie, Alpha Tau VP Quality and Regulatory Affairs, noted, "This amazing milestone represents the culmination of Alpha Tau’s rigorous teamwork and unwavering commitment to quality and regulatory excellence."