- Glioblastoma, also known as GBM, is a fatal and incurable brain cancer that tends to recur aggressively after initial treatment
- CNS Pharmaceuticals is a U.S.-based pharmaceutical innovator whose flagship drug candidate, Berubicin, is a potentially pivotal treatment for treating GBM because of its novel apparent ability to cross the blood-brain barrier at effective levels
- CNS is building upon prior clinical trial outcomes with its global potentially pivotal trial currently registering patients in the United States and Europe
- Berubicin’s development is being advanced with potential time and cost savings thanks to the FDA’s decision to grant it Fast Track and Orphan Drug status
The most common primary brain cancer in adults and one of the most aggressive, glioblastoma, remains a critical unmet medical need because of its level of morbidity and the lack of effective treatment options nearly a century after it was defined by pioneering neurosurgeons Percival Bailey and Harvey Cushing in 1926.
Glioblastoma, known simply as GBM, is effectively 100 percent fatal with extremely rare, noteworthy exceptions. The brain cancer is diagnosed in approximately 13,000 U.S. patients and only 10 percent or less of them survive to five years, most succumbing within about a year (https://ibn.fm/epCgQ).
Biopharmaceutical innovator CNS Pharmaceuticals (NASDAQ: CNSP) is enrolling patients in the United States and Europe for its potentially pivotal global clinical trial in humans for its lead drug candidate designed to treat GBM.
The drug, Berubicin, is an anthracycline chemotherapy agent that apparently has the unique ability to cross the blood-brain barrier to target tumors directly. The inability of anthracyclines to cross the blood-brain barrier has been identified as a major obstacle in treating aggressive brain tumors such as GBM and creates an opportunity for CNS Pharmaceuticals.
“Anthracyclines are designed to damage the DNA in cancer cells,” CEO John Climaco said during a 2020 webinar about the potentially pivotal global trial (https://ibn.fm/6WbPd). “Anthracyclines as a class of chemotherapy have been used for over 60 years to treat a variety of cancers, including breast, ovarian, lung, lymphoma and leukemia, and other malignancies as well. However, historically, anthracyclines have never been used to treat primary or metastatic brain cancers because scientists could not demonstrate that anthracyclines were able to cross the blood-brain barrier and achieve significant levels of activity in the brain. Berubicin may change that history.”
A Nov. 14 news release provided a corporate update, noting that 68 clinical sites have now been identified in the United States, Italy, France, Spain, and Switzerland with 29 of them currently enrolling patients (https://ibn.fm/AtOme). The news release shows several sites have been added even since the U.S. National Library of Medicine’s clinical trials registry was updated earlier this month. The majority of the recruiting sites are located in the United States, spread from California to New York, with sites in France the most recent to begin enrolling patients (https://ibn.fm/xulW0).
The company is taking a phased approach to funding the worldwide complex trial, with current funding sufficient for operations into next year. During the corporate webinar, Chief Medical Officer Dr. Sandra Silberman said up to 243 patients will participate in the trial, 162 of them receiving Berubicin and the other 81 receiving the chemotherapy drug lomustine.
“We are evaluating responses defined as a decrease in the size of the tumor, as well as stability of the disease defined as no further increases in the size of the tumor once the patient is put on study, as well as the time to progression of these tumors and importantly overall survival of the patients,” Silberman said.
Radiation and surgical removal can significantly improve overall survival (“OS”) of GBM patients but because of the tumor’s growth from critical brain tissue, it is generally impossible to completely remove the tumor without harming the patient so the cancer tends to recur.
With recurrent GBM, medical intervention has been able to improve progression-free survival (“PFS”) for a few months but not OS, according to a recent report published in Oncology Nursing News (https://ibn.fm/kXguz). Research presented in a poster at November’s 27th Annual Meeting of the Society for Neuro-Oncology compared treatment efficacy and patient characteristics in phase 3 trials of adults newly diagnosed with glioblastoma between 2005 and 2021, and identified 11 trials of 100-plus patients with OS as an endpoint, of which only three reported an OS benefit ranging between medians of 12 and 48 months (https://ibn.fm/PD0wZ).
Berubicin appeared on the scene as a potential outlier in 2006 when a small safety trial conducted by its original developer, Reata Pharmaceuticals, resulted in one of the two dozen evaluated patients emerging cancer-free, remaining so over the years since, while nearly half of the trial’s other patients experienced a statistically significant improvement in clinical benefit (https://ibn.fm/Ngg2z).
CNS entered a collaboration agreement with Reata after its founding in 2017, leading to the current effort to establish Berubicin’s effectiveness and take it to market.
For more information, visit the company’s website at www.CNSPharma.com.
NOTE TO INVESTORS: The latest news and updates relating to CNSP are available in the company’s newsroom at https://ibn.fm/CNSP
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