CNS Pharmaceuticals Acquires Orphan Drug Designation for Novel, Late Stage Abeotaxane, TPI 287
CNS Pharmaceuticals (NASDAQ:CNSP) has successfully acquired Orphan Drug Designation for TPI 287, a novel abeotaxane drug, from Cortice Biosciences. The FDA previously granted these designations for treating gliomas, pediatric neuroblastoma, and progressive supranuclear palsy.
TPI 287 works similarly to other taxanes by stabilizing microtubules and inhibiting cell division. Unlike most taxanes, clinical data suggests it can cross the blood-brain barrier. In a Phase 1 trial combining TPI 287 with bevacizumab for glioblastoma treatment, 3 Complete Responses and 9 Partial Responses were observed among 23 evaluable patients.
The company plans to begin Phase 2 study enrollment by year-end 2025. The Orphan Drug Designation provides benefits including tax credits, FDA fee exemptions, and potential 7-year market exclusivity post-approval.
CNS Pharmaceuticals (NASDAQ:CNSP) ha ottenuto con successo la Designazione di Farmaco Orfano per TPI 287, un nuovo farmaco abeotaxane, da Cortice Biosciences. La FDA aveva già concesso queste designazioni per il trattamento di gliomi, neuroblastoma pediatrico e paralisi sopranucleare progressiva.
TPI 287 agisce in modo simile ad altri taxani stabilizzando i microtubuli e inibendo la divisione cellulare. A differenza della maggior parte dei taxani, i dati clinici suggeriscono che possa attraversare la barriera emato-encefalica. In uno studio di Fase 1 che combinava TPI 287 con bevacizumab per il trattamento del glioblastoma, sono state osservate 3 risposte complete e 9 risposte parziali su 23 pazienti valutabili.
L'azienda prevede di iniziare l'arruolamento per lo studio di Fase 2 entro la fine del 2025. La Designazione di Farmaco Orfano offre vantaggi quali crediti d'imposta, esenzioni dalle tasse FDA e una possibile esclusività di mercato di 7 anni dopo l'approvazione.
CNS Pharmaceuticals (NASDAQ:CNSP) ha obtenido con éxito la Designación de Medicamento Huérfano para TPI 287, un nuevo fármaco abeotaxano, de Cortice Biosciences. La FDA previamente otorgó estas designaciones para el tratamiento de gliomas, neuroblastoma pediátrico y parálisis supranuclear progresiva.
TPI 287 funciona de manera similar a otros taxanos al estabilizar los microtúbulos e inhibir la división celular. A diferencia de la mayoría de los taxanos, los datos clínicos sugieren que puede cruzar la barrera hematoencefálica. En un ensayo de Fase 1 que combinó TPI 287 con bevacizumab para el tratamiento del glioblastoma, se observaron 3 respuestas completas y 9 respuestas parciales entre 23 pacientes evaluables.
La compañía planea comenzar la inscripción para el estudio de Fase 2 a finales de 2025. La Designación de Medicamento Huérfano ofrece beneficios como créditos fiscales, exenciones de tarifas de la FDA y una posible exclusividad en el mercado de 7 años tras la aprobación.
CNS Pharmaceuticals (NASDAQ:CNSP)는 Cortice Biosciences로부터 새로운 아베오탁산 약물인 TPI 287에 대해 희귀의약품 지정(Orphan Drug Designation)을 성공적으로 획득했습니다. FDA는 이전에 교모세포종, 소아 신경아세포종, 진행성 핵상마비 치료를 위해 이러한 지정을 부여한 바 있습니다.
TPI 287은 다른 탁산 계열 약물과 유사하게 미세소관을 안정화하고 세포 분열을 억제합니다. 대부분의 탁산과 달리 임상 데이터는 이 약물이 혈액뇌장벽을 통과할 수 있음을 시사합니다. 교모세포종 치료를 위한 TPI 287과 베바시주맙 병용 1상 시험에서 23명의 평가 가능한 환자 중 3명의 완전 반응과 9명의 부분 반응이 관찰되었습니다.
회사는 2025년 말까지 2상 시험 등록을 시작할 계획입니다. 희귀의약품 지정은 세금 공제, FDA 수수료 면제, 승인 후 최대 7년간 시장 독점권 등의 혜택을 제공합니다.
CNS Pharmaceuticals (NASDAQ:CNSP) a obtenu avec succès la désignation de médicament orphelin pour TPI 287, un nouveau médicament abeotaxane, auprès de Cortice Biosciences. La FDA avait précédemment accordé ces désignations pour le traitement des gliomes, neuroblastome pédiatrique et paralysie supranucléaire progressive.
TPI 287 agit de manière similaire aux autres taxanes en stabilisant les microtubules et en inhibant la division cellulaire. Contrairement à la plupart des taxanes, les données cliniques suggèrent qu'il peut traverser la barrière hémato-encéphalique. Lors d'un essai de phase 1 combinant TPI 287 avec le bevacizumab pour le traitement du glioblastome, 3 réponses complètes et 9 réponses partielles ont été observées chez 23 patients évaluables.
L'entreprise prévoit de commencer le recrutement pour l'étude de phase 2 d'ici la fin 2025. La désignation de médicament orphelin offre des avantages tels que des crédits d'impôt, des exonérations de frais FDA et une exclusivité commerciale potentielle de 7 ans après approbation.
CNS Pharmaceuticals (NASDAQ:CNSP) hat erfolgreich die Orphan-Drug-Designation für TPI 287, ein neuartiges Abeotaxan-Medikament, von Cortice Biosciences erhalten. Die FDA hatte zuvor diese Designationen für die Behandlung von Gliomen, pädiatrischem Neuroblastom und progressiver supranukleärer Lähmung vergeben.
TPI 287 wirkt ähnlich wie andere Taxane, indem es Mikrotubuli stabilisiert und die Zellteilung hemmt. Im Gegensatz zu den meisten Taxanen deuten klinische Daten darauf hin, dass es die Blut-Hirn-Schranke überwinden kann. In einer Phase-1-Studie, in der TPI 287 mit Bevacizumab zur Behandlung von Glioblastomen kombiniert wurde, wurden bei 23 auswertbaren Patienten 3 komplette und 9 partielle Ansprechraten beobachtet.
Das Unternehmen plant, die Rekrutierung für die Phase-2-Studie bis Ende 2025 zu beginnen. Die Orphan-Drug-Designation bietet Vorteile wie Steuergutschriften, Befreiungen von FDA-Gebühren und eine potenzielle 7-jährige Marktexklusivität nach der Zulassung.
- Successful acquisition of Orphan Drug Designation for TPI 287, providing potential 7-year market exclusivity
- Promising Phase 1 trial results showing 12 out of 23 patients responding to treatment
- Drug's ability to cross the blood-brain barrier, unlike most similar treatments
- Multiple FDA Orphan Drug Designations for different indications (gliomas, pediatric neuroblastoma, and progressive supranuclear palsy)
- Tax credits and FDA fee exemptions from Orphan Drug status
- Phase 2 study enrollment won't begin until year-end 2025, indicating a long development timeline
- Early-stage development status with significant clinical trials still needed
- Targeting rare diseases with small patient populations (fewer than 200,000 people in the U.S.)
Insights
CNS Pharma's acquisition of Orphan Drug Designation for TPI 287 provides regulatory advantages and market exclusivity for a promising GBM treatment.
The acquisition of Orphan Drug Designation (ODD) for TPI 287 from Cortice Biosciences represents a significant regulatory milestone for CNS Pharmaceuticals. This designation brings substantial benefits that enhance the commercial potential of this asset, including tax credits, fee exemptions, and critically, seven years of market exclusivity upon approval.
TPI 287's mechanism differentiates it from other taxanes in a crucial way - while maintaining the same cytotoxic action by stabilizing microtubules and inhibiting cell division, it appears capable of crossing the blood-brain barrier, a major hurdle in treating central nervous system malignancies. This property addresses a fundamental challenge in neuro-oncology therapeutics.
The preliminary clinical data is particularly noteworthy: a Phase 1 trial combining TPI 287 with bevacizumab (Avastin®) demonstrated 3 Complete Responses and 9 Partial Responses among 23 evaluable glioblastoma patients - a response rate of approximately 52%. In the context of GBM, a notoriously aggressive brain cancer with limited treatment options and poor outcomes, these early results suggest meaningful clinical activity.
The company's timeline to initiate Phase 2 enrollment by year-end 2025 indicates steady progression in clinical development. The transfer of multiple ODDs for different indications (gliomas, pediatric neuroblastoma, and progressive supranuclear palsy) creates potential for pipeline expansion across several rare neurological conditions, diversifying the company's development opportunities while maintaining focus on CNS malignancies.
Company advancing plans to develop TPI 287, a novel, late stage abeotaxane that appears to cross the blood-brain barrier and has published clinical efficacy data in glioblastoma multiforme (GBM)
Management releases "What This Means" segment discussing the receipt of Orphan Drug Designations for TPI 287; Access here
HOUSTON, TX / ACCESS Newswire / May 13, 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 successful transfer from Cortice Biosciences, Inc. of Orphan Drug Designation for TPI 287. The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug Designations for TPI 287 in treating gliomas, pediatric neuroblastoma, and progressive supranuclear palsy. Additionally, the Company announced the release of a "What This Means" segment to discuss the Orphan Drug Designations for TPI 287, which is now available here.
TPI 287 is an abeotaxane with the same mechanism of action as other taxanes, such as paclitaxel (Taxol®) and docetaxel; it stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. Although most taxanes are substrates for multi-drug resistant transporters that maintain the blood brain barrier (BBB), TPI 287's clinical data suggest it has the potential to cross the BBB and treat CNS tumors. In a Phase 1 trial treating glioblastoma patients with TPI 287 in combination with bevacizumab (Avastin®), data include 3 Complete Responses and 9 Partial Responses out of 23 patients evaluable.
CNS CEO John Climaco stated, "The successful transfer of the Orphan Drug Designations for TPI 287 demonstrates our operational efficiency as well as our ongoing commitment to the development of neuro oncology-focused, cutting-edge chemotherapies. As we prepare to commence patient enrollment for a Phase 2 study around year-end 2025, this important step also recognizes the significant protection Orphan status may confer in the form of seven years of market exclusivity. Our TPI 287 program is supported by published clinical and radiologic data showing positive activity against GBM, and a favorable safety profile from testing in hundreds of patients. With our demonstrated commitment to defeating this deadly disease, we will be aggressively developing TPI 287 as a treatment option for GBM, where patients continue to face a near-uniformly fatal unmet clinical need."
The FDA Office of Orphan Products Development grants Orphan Drug Designation to products that show the potential to treat rare diseases or conditions, which primarily are those that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies a company for a number of benefits intended to provide incentives to develop drugs for rare diseases or conditions, including tax credits, exemptions from certain FDA fees (which can be significant), and the potential for seven years of market exclusivity following drug approval.
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 drug candidate TPI 287 is an abeotaxane, which stabilizes microtubules and inhibits cell division, causing apoptosis and cell death. The initial clinical efficacy data suggest TPI 287 has the potential to cross the blood-brain barrier and treat CNS tumors. TPI 287 also has been tested in over 350 patients in clinical trials as a monotherapy and in combination with bevacizumab for the treatment of a range of diseases or conditions, including recurrent glioblastoma, recurrent neuroblastoma and medulloblastoma, advanced malignancies, progressive neoplastic disease, advanced unresectable pancreatic cancer, metastatic melanoma, and breast cancer metastatic to the brain. To date TPI 287 appears have both an excellent safety profile and high tolerability among patients.
For more information, please visit www.CNSPharma.com, and connect with the Company on X, Facebook, 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 press release include, without limitation, the timing of the commencement of the Phase 2 study of TPI 287. 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 Contact
JTC Team, LLC
Jenene Thomas
908.824.0775
CNSP@jtcir.com
SOURCE: CNS Pharmaceuticals, Inc.
View the original press release on ACCESS Newswire
FAQ
What is TPI 287 and how does it work in treating brain cancer?
What were the results of CNSP's Phase 1 trial for TPI 287 in glioblastoma patients?
What benefits does the Orphan Drug Designation provide to CNSP's TPI 287?
When will CNS Pharmaceuticals begin Phase 2 trials for TPI 287?
What conditions has TPI 287 received Orphan Drug Designation for?
