CNS Pharmaceuticals is providing hope to those who suffer from one of the deadliest forms of cancer

Glioblastoma multiforme, or GBM, may not sound familiar but if you suffer from this aggressive form of brain cancer the prognosis isn't good. Biopharmaceutical company CNS Pharmaceuticals Inc (NASDAQ:CNSP), though, is looking to change that with its powerful chemotherapy drug Berubicin. On May 19, the company reached an important milestone with its announcement of open enrollment for its potentially pivotal study evaluating the efficacy and safety of Berubicin in the treatment of recurrent GBM. “It's probably the most important thing that we've announced, really, since the founding of the company, because we're now going to have a chance to put this drug, this very helpful drug, back into humans whom we know are in a very bad place,” CNS Pharmaceuticals CEO John Climaco told Proactive recently.   WATCH: CNS Pharmaceuticals begins enrollment for a Phase 2 clinical study to treat aggressive brain cancer The company intends to enroll about 243 subjects across 35 clinical sites in the US and also plans to expand the trial into western Europe.  A pre-planned, non-binding futility analysis will be performed after about 30% to 50% of all planned patients have completed the primary endpoint at six months. This review will include additional evaluation of safety as well as secondary efficacy endpoints. Enrollment will not be paused during this interim analysis. 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, examining the precise biologic transport issue that has prevented efficacy in GBM in the past rather than solely focusing on the tumor itself. Key to developing an effective therapeutic was penetrating the blood brain barrier For 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. What Priebe did was start with a doxorubicin molecule, a drug considered among the most powerful cancer-fighting therapeutics for about 60 years, then modified it by changing its molecular weight and structure to allow it to bypass the blood-brain barrier. “That seemingly small innovation on a molecular level, I think is going to be the key to changing the way GBM is treated,” Climaco said. CNS Pharmaceuticals acquired Berubicin from Reata Pharmaceuticals, Inc (NASDAQ:RETA), which conducted a successful Phase 1 safety testing of the drug before exiting the oncology business. For CNS Pharma, the next step for Berubicin is conducting studies that are focused primarily on demonstrating efficacy to present data for eventual registration and marketing approval. “And that's really where (CNS Pharma) is right now, which is the most exciting part of the development process where you are actually attempting to prove that this drug has a benefit to these patients,” Climaco noted. One patient in the Phase 1 study is still alive 14 years later  While the company’s ultimate goal is to demonstrate efficacy, it is doing so by focusing on its primary endpoint -- extending the life expectancy of people with GBM.      In that regard, CNS Pharma is encouraged by a patient in the Phase 1 study who had what's called a durable complete response, meaning the tumor after treatment was undetectable. And that patient is still alive 14 years later.   In that same Phase 1 study, which had a sample size of 35 patients, 44% of participants had a clinical benefit of stable disease or better, and some other partial responders experienced a tumor reduction of up to 80%. Secondary endpoints of the Berubicin study, meanwhile, include reduction in tumor size, improvement in progression-free survival and quality of life, but Climaco pointed out the company needs to look at the whole picture.  “I approach this from the perspective that the line is set by the US Food and Drug Agency (FDA). So, if overall survival is what gets the drug approved in this space, then that's the key endpoint, that's the primary endpoint,” Climaco added. And although it’s difficult to judge the potential market size for Berubicin, temozolomide is a billion-dollar cancer drug but is only effective in 40% of cases according to Climaco.    He estimates there are about 15,000 cases of primary GBM in the US each year, but suggests if you take into consideration other cancers that spread to the brain that number is much larger. As the second-largest shareholder of CNS Pharma, Climaco’s interests are aligned with the company owners. While as a fellow investor, Climaco said shareholders should expect more news flow about additional Berubicin studies as the company widens its search globally for patients. He pointed out that a potential catalyst for the stock price in the next year could come with interim data from an open-label, single-arm Berubicin study being conducted by its partners in Poland. These results are expected years earlier than some similar US studies, which should provide a preliminary look at how Berubicin is behaving in the same population type.       Finally, given the grim prognosis for those patients diagnosed with GBM, Climaco said CNS Pharma is currently preparing a fast track application for Berubicin with the FDA, a US federal agency he believes is incentivized to see good drugs move through the process as quickly as possible. And, if there were ever an administration that would be the most supportive and promote GBM therapeutics, it would likely be the Biden administration as the US president lost his son to the disease.    CNS Pharmaceuticals, like most developmental-stage drug companies, will need to raise capital to fund its programs, yet Climaco says the company has enough cash currently to last through the first quarter of next year.    Climaco noted that both he and CNS Pharma chief financial officer Chris Downs have large ownership stakes in the company and, as such, are constantly evaluating the most shareholder-friendly ways to finance its growth.    “The way you create lasting shareholder value is by getting drugs approved -- so we want to do that as fast as possible,” Climaco said.  Contact Sean at sean@proactiveinvestors.com