- Biotechnology developer CNS Pharmaceuticals is working to advance the clinical trials of a novel brain cancer-fighting drug candidate with the aim of developing a new, effective therapy for treating an otherwise incurable disease
- The company plans a complex, multi-armed Phase 2 trial of its candidate Berubicin in hopes that the drug candidate may ultimately gain an expedited pathway to approval and registration from the FDA
- The trial for combatting Glioblastoma Multiforme is expected to begin next year, and on Nov. 12 the company’s officers launched a webinar to provide information on how the trial will be designed
- CNS Pharmaceuticals is also preparing, in partnership with its sub-licensee WPD Pharmaceuticals, for two additional trials of Berubicin to be conducted in Poland including the first-ever Phase 1 pediatric trial as well as a parallel Phase 2 trial in adults
A biopharmaceutical company working to find a better way of treating an aggressive form of brain tumor, Glioblastoma Multiforme (“GBM”), which is currently regarded as incurable and ultimately fatal, announced recently that its submission of an Investigational New Drug (“IND”) application to the U.S. Food & Drug Administration (“FDA”) has been accepted for review. It includes a novel clinical trial design it hopes will lead to a breakthrough, and on Nov. 12 company officers discussed the design for the upcoming Phase 2 U.S. trial in a webinar open to the public (https://ibn.fm/bV2e2).
“I would like to remind everyone that this upcoming Phase 2 trial will build on the success of the Phase 1 trial of Berubicin in which the clinical benefit response was 44 percent, including one patient … who had a durable complete response and is still alive and cancer-free today, 14 years after treatment with Berubicin, and another two patients with partial responses, who had reductions of greater than 25 percent in the size of their tumors,” CNS Pharmaceuticals (NASDAQ: CNSP) CEO John Climaco told the webinar audience.
CNS Pharmaceuticals’ lead drug candidate in treating GBM, Berubicin, is an anthracycline.
“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,” Climaco said.
“Berubicin may change that history because it is the first anthracycline that, based on limited clinical data, appears to cross the blood-brain barrier and achieve drug levels critical for efficacy against central nervous system malignancies,” he said.
CMO Dr. Sandra Silberman explained that the Phase 2 trial will allow a real-time comparison between Berubicin patients and patients receiving the normal standard of care, using “interim analyses that could impact and in fact reduce the numbers of patients required to establish the effectiveness of Berubicin.”
Under the parameters of the trial, 243 patients will participate, 162 of them receiving Berubicin and the other 81 receiving the chemotherapy drug lomustine. About 60 study centers will be used in North America, Europe and the Asia-Pacific region. Once 50 percent of the patients have been in the study for six months, the interim analysis will begin.
“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.
The study will also be designed to preserve patient safety even with the continuation of the COVID-19 pandemic and GBM patients who don’t ultimately qualify for the study will be allowed other avenues to try Berubicin under U.S. “right to try” laws.
“We now have a drug supply manufactured and will do our best to get these patients that could benefit from Berubicin and have failed standard therapies (to where they) can be part of these parallel studies for which we will continue to accrue safety and efficacy information,” Silberman said.
Additional information about such opportunities will be posted on the company’s website.
Climaco said the complex trial design is not only the most potentially beneficial for studying Berubicin’s effects, but ultimately the most cost-effective design for the company’s shareholders as well because the Phase I trial not only showed the drug’s potential for safe use, but efficacy also.
“Rather than conduct a single-arm trial of Berubicin that would likely simply show us more interesting positive data, we chose to effectively allow our Phase 2 trial to incorporate an arm receiving standard of care such that any significant impact of Berubicin could be better analyzed,” he said.
Because the trial is expected to eventually cost $30 million to $35 million, Climaco said the company is taking a phased fund-raising approach over the next two to two and a half years, progressing from one success to the next rather than trying to raise all of the capital at once. Sound results from the Phase 2 trial could potentially sway the FDA to grant Berubicin an expedited pathway to approval, which would save time in the long run.
“Which is of course the valuable important commodity here,” he said.
CNS Pharmaceuticals is also preparing, in partnership with its sub-licensee WPD Pharmaceuticals, for two additional trials of Berubicin to be conducted in Poland. The first is a first-ever Phase 1 pediatric trial. The secondi s a parallel Phase 2 trial in adults that will be used as additional data to supplement and strengthen the data submitted to the FDA from the primary trial conducted by CNS Pharmaceuticals.
CNS Pharmaceuticals is also developing a drug candidate known as WP1244 with a DNA-binding agent that preclinical studies have shown to be 500 times more potent than chemotherapy drug daunorubicin in stopping tumor cell expansion. This drug is undergoing additional pre-clinical studies in preparation for a potential future Phase 1 clinical trial.
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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