Glioblastoma Multiforme (GBM) Treatments Advancing as FDA Steps Up Measures Due to Rising Incidences

PALM BEACH, Fla., Dec. 17, 2020 /PRNewswire/ -- Glioblastoma multiforme (GBM) is the most common and aggressive malignant brain tumor in humans that involves glial cells. GBM is present in two variants namely, giant cell glioblastoma and gliosarcoma. Gliomas are tumors arising from glial cells and may occur in the spinal cord or the brain, the latter being more common. Gliomas are the most common type of brain tumor and can be either supratentorial or infratentorial.  Seizure, nausea, headache, hemiparesis and memory loss are some of the common symptoms observed in the patients suffering from GBM. GBM is most commonly found in male with the age of 65 years and above. Increasing habits of alcohol consumption, changing lifestyle and increasing incidence of seizure are some of the drivers for the growth of GBM market. GBM is generally treated by first surgically removing the tumor then treating with chemotherapy and radiation. While this treatment plan is the currently accepted standard of care, it does not effectively prevent tumor recurrence. Primary treatment involves surgery to achieve tumor debulking, followed by a multimodal regimen of radiotherapy and chemotherapy. Chemotherapy can be added to radiotherapy for patients with unfavorable low-grade glioma to improve progression free survival. Active biotech and pharma companies in the markets this week include CNS Pharmaceuticals, Inc. (NASDAQ: CNSP), Sio Gene Therapies Inc. (NASDAQ: SIOX), Heat Biologics, Inc. (NASDAQ: HTBX), Bristol Meyers Squibb (NYSE: BMY), Gilead Sciences, Inc. (NASDAQ: GILD).

According to a report from Grand View Research the global glioblastoma multiforme treatment market is expected to witness significant growth over the forecast period owing to the rising incidence of oncology diseases and rising geriatric population base.  The report continued: "According to U.S. National Cancer Institute GBM is considered as the most aggressive form of brain cancer which represents 15.4% of all primary brain tumors and about 60% - 75% of all astrocytoma and shows rapid growth rate of benign cells in the organ. Currently unavailable therapies and prognosis for the treatment of GBM is expected to impact the market growth positively through to 2022. Presence of the treatment options which do not increase the overall survival rate in patients such as surgical resection, which is followed by chemotherapy and radiotherapy, is another factor attributing towards the expected growth of this market. Moreover, the fact that glioma stem cells resist conventional treatments also raises urgent need for alternative treatment therapies for glioblastoma multiforme thus driving the market. Increasing R&D in gene therapy and molecular biotechnology for the treatment of CNS associated disorders and cancer is anticipated to fuel the market growth over the forecast period."

CNS Pharmaceuticals, Inc. (NASDAQ: CNSP) BREAKING NEWS:  CNS Pharmaceuticals Announces FDA Approval of IND Application for its Brain Cancer Drug Candidate Berubicin - IND Approval Marks Key Step in Beginning of Potentially Pivotal Trial of Berubicin - CNS Pharmaceuticals, a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers of the brain and central nervous system, today announced that the Investigational New Drug (IND) application for its lead product candidate, Berubicin, for the treatment of Glioblastoma Multiforme (GBM) is now approved and in effect as filed with the US Food and Drug Administration (FDA). The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy.  Recent correspondence between the Company and the FDA resulted in modifications to the previously disclosed trial design, including designating overall survival (OS) as the primary endpoint of the study. OS 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.

"Since becoming a public company, our clear focus has been on advancing the clinical development of Berubicin. We will now rapidly move to initiate our Phase 2 trial of Berubicin for adults with GBM and expect to begin enrolling patients in the first quarter of next year," commented John Climaco, CEO of CNS Pharmaceuticals. "The Company will transform within the next several months as Berubicin becomes the subject of up to three active clinical trials, which include our randomized, controlled Phase 2 trial in the U.S., and 2 trials planned by our sublicensee WPD in Poland. We are entering an area with significant unmet medical need since the current treatment paradigm for GBM remains bleak, as this aggressive and currently incurable form of brain cancer continues to claim high mortality rates. We have a tremendous opportunity ahead of us as we continue our mission to improve patient outcomes for GBM and build on the promising results demonstrated by Berubicin in its Phase 1 clinical trial."

As previously announced, the planned trial will evaluate the efficacy of Berubicin in patients with GBM who have failed primary treatment for their disease. Results will compare Berubicin to the current standard of care, with a 2 to 1 randomization of patients to either receive Berubicin or Lomustine. The trial's adaptive design is intended to allow an interim analysis of the data to demonstrate meaningful differences in efficacy between treatments and then to allow an adjustment to the size of the patient population in the trial for maximum efficiency in terms of time in development. Based on this, the trial has the potential to provide data to the FDA that may allow an expedited pathway for development. However, there can be no assurance that the FDA will support any potential request for an expedited pathway to approval or further development. Read this full release and more news for CNSP at:  https://www.financialnewsmedia.com/news-cnsp/     

Other recent developments in the biotech industry include:

Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically improve the lives of patients with neurodegenerative diseases, recently reported positive six-month follow-up data from the low-dose cohort (1.5x10¹³ vg/kg) of the Company's dose escalation study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis. Initial data from the ongoing Phase 1/2 study in five patients in the low-dose cohort showed that AXO-AAV-GM1 was generally well tolerated with a favorable safety profile and provide early indications of clinical disease stability.

"We are excited to report encouraging safety, tolerability, biomarker, and preliminary efficacy data for AXO-AAV-GM1, the first gene therapy evaluated in a clinical trial for GM1 gangliosidosis, a life-limiting disease caused by mutations in the GLB1 gene that impair beta-galactosidase enzyme activity. Safety was the key measure in this first-in-human study, and we are pleased to see a favorable safety profile in the first five children treated with the low-dose," said Gavin Corcoran, M.D., Chief R&D Officer of Sio Gene Therapies. "At this early timepoint post-treatment, we observed an increase in beta-galactosidase enzymatic activity, reaching on average 38% of normal reference levels. This is encouraging given that evidence in the medical literature for lysosomal storage diseases suggests that increases in enzyme activity, to between 10-20% of normal levels, can lead to clearance of stored lysosomal substrates and may be associated with slower disease progression. We are also encouraged by consistent signs of disease stabilization across multiple measures of neurodevelopment in all five children at six months as compared to the predictable decline observed in natural history studies. These data highlight the potential for the investigational gene therapy to treat the underlying genetic cause of this disease, preserve functional outcomes, and reduce disease burden for patients and their families. Further, the safety profile observed in the low-dose cohort support moving forward with the high-dose cohort of AXO-AAV-GM1 in the ongoing Phase 1/2 study. We look forward to presenting program updates at future medical conferences."

Heat Biologics, Inc. (NASDAQ: HTBX), a clinical-stage biopharmaceutical company focused on developing first-in-class therapies to modulate the immune system, including multiple oncology product candidates and a novel COVID-19 vaccine, recently announced it has completed its gp96-based COVID-19 vaccine cell line ("ZVX-60"), which is being developed for use as either a standalone vaccine, or in combination with other vaccines to enhance prophylactic protection. Data, generated at the University of Miami Miller School of Medicine, has confirmed expression of gp96, OX40L and Spike protein. The Company also reports it has begun preparations for manufacturing, as the next-step towards first-in-human Phase 1 clinical trials.

Jeff Wolf, Chief Executive Officer of Heat, commented, "We are encouraged by the recent Phase 3 data reported by other vaccine developers, and commend the FDA for their fast action to accelerate approval. Importantly, we believe ZVX-60 holds promise as an adjunct therapy to enhance protection provided by other vaccine approaches. Our preclinical data thus far suggests that our cell line generates a robust T-cell mediated immune response directed against the Spike protein of SARS-CoV-2. Notably, the cell line induced expansion of both "killer" CD8+ T-cells that destroy virus infected cells, as well as "helper" CD4 T-cells that assist in producing anti-viral antibodies."

Bristol Meyers Squibb (NYSE: BMY) recently announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Inrebic^®(fedratinib) for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis, who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. The CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Inrebic will be the first, once-daily oral therapy to significantly reduce spleen volume and symptom burden for patients with myelofibrosis where treatment with ruxolitinib has failed or who are JAK inhibitor naïve.

The CHMP adopted a positive opinion based on results from the JAKARTA and JAKARTA2 studies. The pivotal JAKARTA study evaluated the efficacy of once-daily oral doses of Inrebic compared with placebo in 289 patients with intermediate-2 or high-risk primary or secondary myelofibrosis with splenomegaly. The JAKARTA2 study evaluated the efficacy of once-daily oral doses of Inrebic in 97 patients with intermediate or high-risk primary or secondary myelofibrosis with splenomegaly previously treated with ruxolitinib.

Gilead Sciences, Inc. (NASDAQ: GILD) recently presented new data from the Phase 3 ASCENT trial of Trodelvy^® (sacituzumab govitecan-hziy) in metastatic triple-negative breast cancer (mTNBC) at the 2020 San Antonio Breast Cancer Symposium being held virtually December 8-11, 2020. The new data and analyses from the ASCENT trial continue to demonstrate the high clinical activity of Trodelvy in this patient population with traditionally poor outcomes.

Trodelvy is a Trop-2-directed antibody and topoisomerase inhibitor conjugate that is indicated in the U.S. for the treatment of adult patients with mTNBC who have received at least two prior therapies for metastatic disease. Trodelvy received accelerated approval for this patient population in April 2020, based on objective response rate and duration of response results observed in a single-arm, multicenter Phase 2 study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. The Trodelvy U.S. Prescribing Information has a BOXED WARNING for severe neutropenia and severe diarrhea; see below for Important Safety Information.

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