Glioblastoma multiforme (GBM) is an aggressive and deadly form of brain cancer. For those afflicted, the prognosis is bleak but biopharmaceutical company CNS Pharmaceuticals (NASDAQ:CNSP) Inc is providing hope with the progress the company has been making in its clinical trials involving the powerful novel chemotherapy drug Berubicin.
On September 30, 2021, CNS Pharma announced an important milestone – it began dosing the first patients with Berubicin in its potentially pivotal clinical trial. The company plans to enroll approximately 243 individuals with GBM at clinical trial sites in the US and throughout Europe.
“I think a lot of pieces have come together really nicely for the company,” CNS Pharmaceuticals (NASDAQ:CNSP) CEO John Climaco told Proactive recently.
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“Getting patients on the drug and moving forward with the study is probably the biggest single thing because, of course, that's the heart and soul of what we're doing, executing this potentially pivotal clinical trial for our study of Berubicin for the treatment of glioblastoma,” Climaco added.
Berubicin was developed by Dr Waldemar Priebe, professor of medicinal chemistry at The University of Texas MD Anderson Cancer Center, who took a different approach to attacking GBM than others. Dr Priebe examined the precise biologic transport mechanisms that have prevented other drugs from having efficacy against GBM in the past.
For Dr Priebe, the key to developing an effective therapeutic was penetrating the blood-brain barrier, a specialized network of cells that surround the brain and creates an active and passive barrier that protects the organ. These cells act as pumping mechanisms, expelling chemotherapy drugs out of the brain faster than the heart can pump them in.
With the first patients having been dosed with Berubicin, the company is now looking forward to an interim analysis
Dr Priebe began with a doxorubicin molecule, a drug considered among the most powerful and widely-used cancer-fighting therapeutics developed in the last 60 years. He then modified this well-understood and clinically-trusted drug by changing its molecular weight and structure to allow it to bypass the blood-brain barrier.
Now with the first patients in the CNS Pharma clinical trial having been dosed with Berubicin, the company looks forward to a pre-planned, non-binding interim analysis. This examination of Berubin’s effectiveness will be performed by the company after 50% of all planned patients have completed six months of treatment.
The key data point to be calculated will be an evaluation of overall survival; in other words, are patients being treated with Berubicin living longer with the therapy than they would have otherwise. This review will include additional evaluation of safety as well as secondary efficacy endpoints that include reduction in tumor size, improvement in progression-free survival, and quality of life. Enrollment will not be paused during this interim analysis.
CNS Pharma noted that in the Phase 1 clinical trial previously conducted evaluating Berubicin in patients with previously treated disease, 44% of the patients showed clinical benefit and one had what's called a durable complete response, meaning the tumor after treatment was undetectable. And that patient is still alive 14 years later. The average life expectancy of patients from this population group is 14.6 months from diagnosis.
The company said overall survival is an endpoint the US Food and Drug Administration (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.
In fact, the FDA recently granted CNS Pharma Fast Track Designation for Berubicin, which the company said enables more frequent interactions with the US government agency to expedite the development and review process in addition to potential approval and commercialization.
“That gives us a potential accelerated pathway toward regulatory approval, giving us some priority review and a higher attention profile at FDA,” Climaco said.
Catalysts for the company’s stock price include a rapid acceleration in patient recruitment
CNS Pharma has also received Orphan Drug Designation from the FDA, which the company noted may provide seven years of marketing exclusivity upon approval of a New Drug Application (NDA).
For CNS Pharma shareholders, catalysts for the company’s stock price could come from a rapid acceleration in patient recruitment going into this quarter and into the beginning of next year as well as interim analysis of data from the Berubicin clinical trial.
“I think investors should look to 2022 as a time when they're going to see some signal as to how Berubicin is doing,” Climaco said.
“And, based on what we know of Berubicin and from the very impressive Phase 1 data, we believe that's going to be a very strong signal in the positive direction,” Climaco added.
In addition to the Berubicin trial, CNS Pharma has a second promising program in its pipeline, which is the development of its WP1874 drug technology that utilizes anthracycline and distamycin-based scaffolds to create small molecule agents and is believed to be 500 times more potent than daunorubicin in inhibiting tumor cell proliferation.
CNS Pharma noted that preclinical studies of WP1874 demonstrated high uptake in the brain with anti-tumor activity, and the company is evaluating the use of WP1874 in the treatment of brain cancers, pancreatic, ovarian, and lymphomas.
“Our investors should look for more news on that and continuing news on the development of our trial and the expansion of our clinical network and recruitment of patients,” Climaco concluded.
Contact Sean at sean@proactiveinvestors.com