CNS Pharmaceuticals Announces Primary Analysis of Berubicin in Second Line Treatment of Glioblastoma Multiforme
CNS Pharmaceuticals (NASDAQ:CNSP) has announced the primary analysis of its clinical trial for Berubicin, an anthracycline treatment for recurrent Glioblastoma Multiforme (GBM). The study compared Berubicin to Lomustine, a standard GBM treatment.
Key findings show that while Berubicin did not demonstrate statistically significant superiority in overall survival (the primary endpoint), it showed comparable clinical outcomes to Lomustine across multiple endpoints. Notably, Berubicin displayed no cardiotoxicity, a common limitation with other anthracyclines, and maintained a favorable safety profile.
The drug is significant as the first anthracycline proven to extensively cross the blood-brain barrier. The company is continuing analysis of outcomes, including advanced imaging review and clinical endpoints. Additionally, CNS Pharmaceuticals is developing TPI 287, a novel taxane with published clinical efficacy data in GBM treatment.
CNS Pharmaceuticals (NASDAQ:CNSP) ha annunciato l'analisi primaria del suo studio clinico per Berubicin, un trattamento con antracicline per il Glioblastoma Multiforme (GBM) ricorrente. Lo studio ha confrontato Berubicin con Lomustina, un trattamento standard per il GBM.
I risultati chiave mostrano che, sebbene Berubicin non abbia dimostrato una superiorità statisticamente significativa nella sopravvivenza globale (l'endpoint primario), ha mostrato risultati clinici comparabili a quelli della Lomustina su più endpoint. È importante notare che Berubicin non ha mostrato cardiotossicità, una limitazione comune con altre antracicline, e ha mantenuto un profilo di sicurezza favorevole.
Il farmaco è significativo in quanto è la prima antraciclina dimostrata in grado di attraversare ampiamente la barriera emato-encefalica. L'azienda sta continuando l'analisi dei risultati, inclusa la revisione delle immagini avanzate e degli endpoint clinici. Inoltre, CNS Pharmaceuticals sta sviluppando TPI 287, un nuovo taxano con dati di efficacia clinica pubblicati nel trattamento del GBM.
CNS Pharmaceuticals (NASDAQ:CNSP) ha anunciado el análisis primario de su ensayo clínico para Berubicin, un tratamiento con antraciclinas para el Glioblastoma Multiforme (GBM) recurrente. El estudio comparó Berubicin con Lomustina, un tratamiento estándar para el GBM.
Los hallazgos clave muestran que, aunque Berubicin no demostró una superioridad estadísticamente significativa en la supervivencia general (el objetivo primario), mostró resultados clínicos comparables a los de Lomustina en múltiples objetivos. Cabe destacar que Berubicin no mostró cardiotoxicidad, una limitación común con otras antraciclinas, y mantuvo un perfil de seguridad favorable.
El medicamento es significativo ya que es la primera antraciclina que se ha demostrado que cruza extensamente la barrera hematoencefálica. La empresa continúa analizando los resultados, incluida la revisión de imágenes avanzadas y los objetivos clínicos. Además, CNS Pharmaceuticals está desarrollando TPI 287, un nuevo taxano con datos de eficacia clínica publicados en el tratamiento del GBM.
CNS Pharmaceuticals (NASDAQ:CNSP)는 재발성 다형성 교모세포종(GBM)을 위한 항암제 Berubicin의 임상 시험 주요 분석 결과를 발표했습니다. 이 연구는 Berubicin과 GBM의 표준 치료제인 로무스틴(Lomustine)을 비교했습니다.
주요 결과는 Berubicin이 전체 생존율(주요 목표)에서 통계적으로 유의미한 우위를 보여주지 않았지만, 여러 목표에서 로무스틴과 비교 가능한 임상 결과를 보였다는 것입니다. 특히, Berubicin은 다른 항암제에서 흔히 나타나는 심장독성을 보이지 않았으며, 안전성 프로필이 우수한 것으로 나타났습니다.
이 약물은 혈액-뇌 장벽을 광범위하게 통과할 수 있는 첫 번째 항암제로서 중요합니다. 회사는 고급 이미징 검토 및 임상 목표를 포함한 결과 분석을 계속하고 있습니다. 또한 CNS Pharmaceuticals는 GBM 치료에서 임상 효능 데이터가 발표된 새로운 탁산 TPI 287을 개발하고 있습니다.
CNS Pharmaceuticals (NASDAQ:CNSP) a annoncé l'analyse principale de son essai clinique pour Berubicin, un traitement à base d'anthracyclines pour le glioblastome multiforme récurrent (GBM). L'étude a comparé Berubicin à la Lomustine, un traitement standard pour le GBM.
Les résultats clés montrent que, bien que Berubicin n'ait pas démontré une supériorité statistiquement significative en termes de survie globale (l'objectif principal), il a montré des résultats cliniques comparables à ceux de la Lomustine sur plusieurs critères. Il est à noter que Berubicin n'a présenté aucune cardiotoxicité, une limitation courante avec d'autres anthracyclines, et a maintenu un profil de sécurité favorable.
Ce médicament est significatif car il s'agit de la première anthracycline prouvée capable de franchir largement la barrière hémato-encéphalique. L'entreprise continue d'analyser les résultats, y compris l'examen d'imagerie avancée et les critères cliniques. De plus, CNS Pharmaceuticals développe le TPI 287, un nouveau taxane avec des données d'efficacité clinique publiées dans le traitement du GBM.
CNS Pharmaceuticals (NASDAQ:CNSP) hat die primäre Analyse seiner klinischen Studie zu Berubicin, einer Anthracyclin-Behandlung für das rezidivierende Glioblastoma Multiforme (GBM), bekannt gegeben. Die Studie verglich Berubicin mit Lomustin, einer Standardbehandlung für GBM.
Wichtige Ergebnisse zeigen, dass Berubicin zwar keine statistisch signifikante Überlegenheit in Bezug auf die Gesamtüberlebensrate (das primäre Ziel) nachweisen konnte, jedoch vergleichbare klinische Ergebnisse zu Lomustin in mehreren Endpunkten zeigte. Bemerkenswert ist, dass Berubicin keine Kardiotoxizität aufwies, eine häufige Einschränkung bei anderen Anthracyclinen, und ein günstiges Sicherheitsprofil aufrechterhielt.
Das Medikament ist bedeutend, da es die erste Anthracyclin ist, die nachweislich die Blut-Hirn-Schranke umfassend überqueren kann. Das Unternehmen setzt die Analyse der Ergebnisse fort, einschließlich der Überprüfung fortschrittlicher Bildgebung und klinischer Endpunkte. Darüber hinaus entwickelt CNS Pharmaceuticals TPI 287, ein neuartiges Taxan mit veröffentlichten klinischen Wirksamkeitsdaten zur Behandlung von GBM.
- First anthracycline to cross blood-brain barrier with no cardiac toxicity
- Favorable safety profile compared to Lomustine with fewer side effects
- Comparable clinical outcomes to current standard of care (Lomustine)
- Potential broader application due to mechanism of action being tumor histology-agnostic
- Failed to meet primary endpoint of superior overall survival
- Did not achieve statistical significance in efficacy compared to existing treatment
Insights
CNS Pharmaceuticals' announcement on Berubicin represents a significant clinical setback for their lead asset. The trial failed to demonstrate statistically significant superiority in overall survival compared to Lomustine, which was the primary endpoint for this GBM study. This outcome substantially diminishes Berubicin's regulatory pathway forward.
While the company highlights that Berubicin showed comparable efficacy to Lomustine across multiple endpoints and demonstrated no cardiotoxicity (a common limitation with anthracyclines), these positive aspects don't overcome the fundamental issue of missing the primary endpoint. Regulatory agencies typically require meeting pre-specified primary endpoints for approval, particularly in oncology.
The favorable safety profile - notably the absence of anthracycline-related cardiotoxicity and fewer pulmonary/hematological adverse events compared to Lomustine - does preserve some value proposition. However, without demonstrating superiority or formally proving non-inferiority (which the trial wasn't powered to do), Berubicin faces significant commercial and regulatory hurdles.
The company's pivot to emphasizing TPI 287, their taxane derivative with published activity in GBM, signals a potential strategic shift following these disappointing results. For a company with
This announcement represents a material negative development for CNS Pharmaceuticals. Failing to achieve statistical significance on the primary endpoint of overall survival dramatically reduces Berubicin's commercial potential and pathway to market. For a micro-cap company (
The company's emphasis on comparable clinical outcomes and safety advantages appears to be setting up potential fallback positions: either pursuing a non-inferiority claim in subsequent trials, exploring specific patient subgroups where benefit might be more pronounced, or repositioning Berubicin as a safer alternative to Lomustine rather than a superior one.
Particularly concerning is that this was a second-line treatment study - typically a lower regulatory hurdle than first-line therapy. The company's pivot to highlighting TPI 287 suggests internal recognition that Berubicin's path forward may be without significant additional investment in larger trials.
While the lack of cardiotoxicity maintains some differentiation for Berubicin, the fundamental value proposition to payers and clinicians has weakened considerably. Small oncology-focused biotechs that miss primary endpoints typically experience significant stock pressure as capital requirements increase while probability of success decreases. CNSP now faces critical strategic decisions regarding pipeline prioritization, potential partnerships, or additional financing that will likely be on less favorable terms given these results.
Berubicin showed clinically relevant outcomes comparable to Lomustine across multiple endpoints, but not a statistically significant difference in overall survival, the primary endpoint.
The safety profile continues to be favorable, including the absence of anthracycline related cardiotoxicity.
Analysis of outcomes are ongoing, including advanced imaging review, PK, and clinical endpoints.
The company also continues development of a novel taxane, TPI 287, with published clinical efficacy data in Glioblastoma Multiforme.
HOUSTON, TX / ACCESS Newswire / March 25, 2025 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP) ("CNS" or the "Company"), a biopharmaceutical company specializing in the development of novel treatments for primary and metastatic cancers in the brain and central nervous system, today announced the primary analysis of its clinical trial (NCT04762069) evaluating Berubicin, the first anthracycline demonstrated to extensively cross the blood-brain barrier (BBB), for the treatment of recurrent or progressive Glioblastoma Multiforme (GBM), an aggressive and usually fatal brain cancer. This analysis did not demonstrate statistically significant superiority in overall survival, the primary endpoint. However, although the trial was not powered to determine non-inferiority, the data appear comparable in clinically important endpoints in all patients, including those with the most unfavorable tumor markers. Additionally, patients experienced no cardiotoxicity, a risk that curtails the use of other anthracyclines, and the safety profile continues to be favorable in this patient population.
The trial compared Berubicin to Lomustine, a current standard of care in recurrent or progressive GBM. The protocol design incorporated the importance of patient phenotype, utilizing well recognized biostatistical techniques in both a pre-specified futility analysis and this analysis. The data appear comparable to Lomustine in important clinically relevant endpoints, including overall survival (OS) and progression free survival (PFS) across all patients treated with Berubicin. Further, the aggregate data facilitate multiple additional clinical analyses for informed hypothesis generation.
"Clinical results in a prior Phase 1 trial indicated Berubicin's potential to improve outcomes for patients with previously treated Glioblastoma," said Sandra Silberman, MD, PhD, Chief Medical Officer of CNS Pharmaceuticals. "In this analysis overall survival is comparable between Berubicin and Lomustine. We are awaiting long-term follow-up of patients still alive as well as those still on trial, and will evaluate our substantial clinical dataset to obtain additional insights. Given the persistent unmet clinical need in GBM and other brain cancers, the results we've seen with Berubicin in this study warrant further investigation of this drug."
Erin Dunbar, MD, a Principal Investigator and the Director of the Piedmont Brain Tumor Center in Atlanta, Georgia noted, "As the longest accruing Investigator, I have witnessed rapid imaging responses and excellent clinical tolerability throughout the trial. I hope to see continued investigation of Berubicin to further evaluate its potential as an important tool to treat primary and/or metastatic cancer patients of all ages who urgently need additional options."
Berubicin is a novel anthracycline, the first to readily penetrate the blood brain barrier and show no cardiac toxicity, thus overcoming two limitations to the use of one of the most effective classes of drugs for the treatment of cancer. "Because of the cardiotoxicity associated with other anthracyclines," Dr. Silberman said, "the fact that this study showed no cardiotoxicity with Berubicin, even in those receiving the drug for over a year, suggests Berubicin could be a valuable recourse for patients with recurrent or progressive cancers." She added, "Berubicin also did not exhibit the pulmonary toxicity or the thrombocytopenia associated with Lomustine, suggesting it could be a way for patients to avoid those side effects during treatment. Furthermore, its primary mechanism of action, topoisomerase II inhibition, is agnostic to specific tumor histology, making it a potentially more generalizable therapy."
CNS Chief Executive Officer John Climaco, JD further added, "We are grateful for the commitment and dedication of patients, caregivers, and investigators who supported this program. We are exploring further Berubicin development as well as evaluating opportunities for applying the methodology and strategy from this program to other drugs for CNS malignancies, including our proprietary drug TPI 287. TPI 287 is a taxane-derivative with published clinical and radiologic data showing activity in Glioblastoma Multiforme, as well as a favorable safety profile in hundreds of patients to date."
About the Berubicin Trial
This ongoing study of Berubicin is a multicenter, open-label, randomized controlled study in adult patients with recurrent GBM (WHO Grade IV - IDH Wild-Type), comparing Berubicin to Lomustine, an accepted treatment for second line therapy in this disease. The study has included 252 patients across North America and Europe, randomizing Berubicin to Lomustine 2:1. Continuation of patients on treatment and their follow-up for overall survival is proceeding. Final data will be included in a future analysis.
For more information about this trial, visit clinicaltrials.gov and reference identifier NCT04762069.
About Berubicin
Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of primary and metastatic cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Berubicin treatment of brain cancer patients appeared to demonstrate positive responses that include one durable complete response in a Phase 1 human clinical trial conducted by Reata Pharmaceuticals, Inc. Berubicin was developed by Dr. Waldemar Priebe, Professor of Medicinal Chemistry at The University of Texas MD Anderson Cancer Center.
About CNS Pharmaceuticals, Inc.
CNS Pharmaceuticals is a clinical-stage pharmaceutical company developing a pipeline of anti-cancer drug candidates for the treatment of primary and metastatic cancers of the brain and central nervous system.
The Company's lead drug candidate, Berubicin, is the first anthracycline to appear to cross the blood-brain barrier. Berubicin is the subject of the Company's late-stage, fully-enrolled, global clinical trial for the treatment of glioblastoma multiforme (GBM), an aggressive and currently incurable form of brain cancer.
The Company's second drug candidate, TPI 287, is an abeotaxane which stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. Similar to Berubicin, TPI 287 has shown the potential to cross the blood-brain barrier and treat CNS tumors. TPI 287 has been well tolerated in over 350 patients to date, including in clinical trials as a monotherapy and in combination with bevacizumab for the treatment of recurrent neuroblastoma and medulloblastoma, as well as refractory prostate cancer and melanoma, and in tauopathy disease, which can result in dementia.
For more information, please visit www.CNSPharma.com, and connect with the Company on X and LinkedIn.
Forward-Looking Statements
Some of the statements in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this release include, without limitation, the results of any future investigation of Berubicin and the release of the final data and analysis from the clinical trial. These statements relate to future events, future expectations, plans and prospects. Although CNS believes the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. CNS has attempted to identify forward-looking statements by terminology including ''believes,'' ''estimates,'' ''anticipates,'' ''expects,'' ''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,'' ''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, including market and other conditions and those discussed under Item 1A. "Risk Factors" in CNS's most recently filed Form 10-K filed with the Securities and Exchange Commission ("SEC") and updated from time to time in its Form 10-Q filings and in its other public filings with the SEC. Any forward-looking statements contained in this press release speak only as of its date. CNS undertakes no obligation to update any forward-looking statements contained in this press release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events, except as required by law.
CONTACTS:
Investor Relations:
JTC Team, LLC
Jenene Thomas
908.824.0775
CNSP@jtcir.com
Media:
Madelin Hawtin
mhawtin@lifescicomms.com
SOURCE: CNS Pharmaceuticals, Inc.
View the original press release on ACCESS Newswire
FAQ
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