- Glioblastoma multiforme (“GBM”) is an aggressive brain cancer with limited treatment options, and CEO John Climaco emphasized the company’s commitment to overcoming the blood-brain barrier, a key obstacle in brain cancer therapy.
- Lead candidate TPI 287, an abeotaxane, has shown early evidence of appearing to cross the blood-brain barrier and producing clinical responses.
- Phase 2 planning is underway following encouraging data from a Phase 1 combination study with bevacizumab (Avastin).
- The company’s repurposed global clinical network and regulatory progress position it for potential late-stage development in 2026.
- TPI 287 holds multiple Orphan Drug Designations, covering gliomas, neuroblastoma, and other CNS-related indications.
CNS Pharmaceuticals (NASDAQ: CNSP), a biopharmaceutical company specializing in novel treatments for primary and metastatic brain cancers is sharpening its focus on glioblastoma multiforme (“GBM”), one of the deadliest and least treatable forms of cancer. In a recent interview on The BioMedWire Podcast, CEO John Climaco described glioblastoma as “one of the two greatest unmet needs in oncology today,” alongside pancreatic cancer (https://ibn.fm/nEVWs).
“There is no cure,” Climaco said. “Patients have very little hope. Our mission is to change that completely, and we think we have the drugs to do it.”
The company’s therapeutic strategy centers on overcoming the blood-brain barrier (“BBB”), a critical biological defense that restricts most drugs from reaching brain tumors. “The problem for patients and clinicians in the space is that the cancers take place in the brain behind the blood-brain barrier, which is this very specialized network of cells that prevents otherwise helpful drugs from reaching the site of the cancer.… We believe that is the reason why a cancer like glioblastoma remains one of the two greatest unmet needs in oncology today, the other being pancreatic cancer,” the CNS Pharmaceuticals CEO explained.
Many promising cancer treatments fail to achieve effective concentrations inside the central nervous system. CNS Pharmaceuticals’ drug candidate, TPI 287, aims to bypass this limitation. TPI 287 belongs to the taxane family, which includes paclitaxel and docetaxel, but is designed to evade drug transporters that typically block these agents at the BBB. The compound works by stabilizing microtubules, preventing cancer cell division and promoting apoptosis.
Early clinical data suggest that TPI 287 could represent progress in treating GBM. In a Phase 1 study combining TPI 287 with bevacizumab (Avastin), 23 patients with recurrent glioblastoma were evaluated. Investigators reported three complete responses and nine partial responses, results that are notable given the disease’s typically poor prognosis.
Median survival for glioblastoma patients remains approximately 15 months despite surgery, radiation, and chemotherapy. As such, any evidence of durable responses in late-stage disease provides valuable insight into potential therapeutic activity.
Climaco also highlighted how the company adapted its operations following earlier work with berubicin, another brain cancer candidate. “When you have a drug development company, you’ve got to realize that 90% of oncology drug trials fail. Those are the odds that you’re facing… That doesn’t dissuade us at all, because we know these drugs work. We have to prove that; that is our job,” he explained. “So, when we pivoted from our berubicin study to our TPI 287 program, we didn’t miss a beat. We have repurposed all of the work that we did to develop a global clinical network for berubicin over to our TPI program. We really just seamlessly moved forward on that.”
This repurposing of existing resources, clinical sites, data systems, and regulatory frameworks, positions CNS Pharmaceuticals to move efficiently toward its next development phase.
CNS Pharmaceuticals recently announced that it will meet with the U.S. Food and Drug Administration later this year to discuss the design of a potential registration-directed Phase 2 study in recurrent GBM. The company anticipates launching the trial in the first half of 2026.
TPI 287 already holds Orphan Drug Designation from the FDA for multiple indications, including gliomas, pediatric neuroblastoma, and progressive supranuclear palsy. These designations can provide financial incentives, fee waivers, and potential market exclusivity upon approval.
In parallel, CNS Pharmaceuticals continues to maintain visibility within the investment and scientific communities. The company presented recent updates at the H.C. Wainwright 27th Annual Global Investment Conference in New York in September 2025, where executives met with institutional investors and analysts. A webcast of the presentation is available on the company’s Events page (https://ibn.fm/kskOJ).
Beyond primary brain tumors, TPI 287 may also have applications in metastatic cancers that spread to the brain. Climaco noted that approximately 40% of patients with triple-negative breast cancer develop brain metastases, representing a significant opportunity for CNS Pharmaceuticals’ drug candidates.
“When you look at the opportunities in the space that a drug like berubicin or TPI 287 could have, not just in the primary brain cancer space, but in the metastatic space, in triple-negative breast cancer – where 40% of patients have a metastasis to the brain – the multiple and the size of the potential market for these drugs is absolutely enormous,” he said. “We have demonstrated what I believe is absolutely world-class operational expertise and the ability to bring a trial through to completion on time and on budget. We’ll do it again with TPI, and we hope that we’ll have a positive result and be on our way to a drug approval.”
So far, TPI 287 has been tested in more than 350 patients across several cancer types, either as a single agent or in combination therapies. These include recurrent neuroblastoma, medulloblastoma, advanced pancreatic cancer, metastatic melanoma, and breast cancer metastatic to the brain. The drug candidate has shown a favorable safety and tolerability profile, which is a crucial factor as CNS Pharmaceuticals moves toward larger clinical studies.
For more information about the company, please visit 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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