Brain Cancer Research: The Race for Better CNS Cancer Treatments Could Open a Multibillion-Dollar Market

Brain Cancer Research: The Race for Better CNS Cancer Treatments Could Open a Multibillion-Dollar Market Brain Cancer Research: The Race for Better CNS Cancer Treatments Could Open a Multibillion-Dollar Market Growing investment in next-generation CNS cancer therapies is driving research into targeted drugs, immunotherapy and new approaches to treating aggressive brain tumors. GlobeNewswire October 21, 2026

NEW YORK, Oct. 21, 2026 (GLOBE NEWSWIRE) -- Brain cancer remains one of the hardest cancers to treat, and researchers are under pressure to find therapies that do more than slow the disease for a short time. For patients with aggressive tumors such as glioblastoma, treatment options remain limited, especially when cancer returns or stops responding to existing drugs. That is why drug developers are exploring new approaches, including targeted therapies, immunotherapy and advanced drug-delivery systems designed to get medicine across the blood-brain barrier. The market opportunity is growing alongside that research. The global brain cancer therapeutics market was estimated at approximately $3.3 billion in 2024, with projections putting it at around $5.1 billion by 2030, according to industry research from MarkNtel Advisors. Better treatments are desperately needed, and a therapy that can meaningfully extend survival or improve quality of life could make a real difference for patients while opening a valuable commercial opportunity.  Active Companies advancing next-generation biotech activities this week include NeOnc Technologies Holdings, Inc. (NASDAQ: NTHI), Neurocrine Biosciences, Inc. (NASDAQ: NBIX), Acadia Pharmaceuticals Inc. (NASDAQ: ACAD), Anavex Life Sciences Corp. (NASDAQ: AVXL), Merck (MSD) (NYSE: MRK).

The science is moving forward, but turning promising research into an approved treatment is still a difficult road. Brain tumors are complex, patients can respond differently to the same drug, and many therapies struggle to reach cancer cells inside the brain. Researchers are working to address those problems through more personalized treatment strategies, molecular testing, immune-based therapies and combinations of existing and emerging drugs. One industry forecast estimates that the global brain tumor therapeutics market could rise from approximately $3.87 billion in 2026 to $6.99 billion by 2032. Those projections point to a market attracting growing interest, but the real turning point will come from clinical results. Companies that demonstrate a treatment can safely reach its target and deliver meaningful benefits may have opportunities for partnerships, licensing deals and eventual commercialization. Clinical trials can fail, regulatory approval is never guaranteed, and development costs can be substantial, but the medical need is clear.

Key Drivers for CNS Cancer Research that is Gaining Attention:

Market projections are estimates, not guarantees. Actual growth will depend on clinical results, regulatory decisions, treatment adoption and commercial execution.

NeOnc Technologies and the Department of Health – Abu Dhabi Sign MoU to Advance Brain Cancer Research and Proposed Clinical Collaboration

NeOnc Technologies Holdings, Inc. (Nasdaq: NTHI) (“NeOnc” or the “Company”), a clinical-stage biopharmaceutical company developing therapies for central nervous system (CNS) cancers, today announced that it has signed a Memorandum of Understanding (MoU) with the Department of Health – Abu Dhabi (DoH) to cooperate on the development of research and clinical capability in oncology and central nervous system therapeutics in the Emirate of Abu Dhabi. The MoU was signed on the sidelines of the Abu Dhabi Future Health Summit (ADFHS) 2026.

Amir F. Heshmatpour, Executive Chairman, President and Chief Executive Officer of NeOnc Technologies Holdings, Inc., said: “For families facing brain cancer, access to investigational treatments and clinical research opportunities matters deeply. This proposed collaboration creates a framework to bring NeOnc’s development expertise together with Abu Dhabi’s clinical capabilities, with the goal of advancing research and expanding opportunities for patients closer to home. We are grateful to the Department of Health – Abu Dhabi for its commitment to addressing the needs of patients with complex and rare CNS cancers, including children.”

H.E. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi (DoH), said: “For patients and families facing complex CNS cancers, every advance in research offers hope for more effective care and better quality of life. Our proposed collaboration with NeOnc connects international expertise with Abu Dhabi’s clinical capabilities to strengthen the evidence and skills needed to evaluate new approaches responsibly. By developing specialist knowledge and a deeper understanding of these critical conditions, we aim to support care that responds more closely to each patient’s needs, reflecting our commitment to bringing scientific advances closer to our community and beyond.”

The MoU builds on NeOnc’s existing activity in the Emirate, where DoH is the government entity responsible for regulating the health sector. In June 2026, DoH granted Investigational New Drug (IND) status for NEO100, covering the Company’s NEO100-01, NEO100-02 and NEO100-03 protocols across Phase 1, Phase 1b and Phase 2 studies in adults with progressive or recurrent Grade III or IV gliomas, together with pediatric studies authorized for Phase 1 and Phase 1b pending further protocol review. That authorization followed DoH’s IND clearance for NEO212, also announced in June 2026. The DoH clearances apply to the conduct of clinical research and do not constitute marketing authorization, and NeOnc must satisfy additional conditions, including separate DoH Institutional Review Board approval, before patient enrollment in the UAE. Two children in Abu Dhabi are currently receiving investigational NEO100 through NeOnc’s Compassionate Care program. A third child is expected to be onboarded from India this week. Building on that experience and on the Company’s existing DoH clearances, NeOnc is working to open a Phase 1 study of NEO100 in the Emirate.

Under the MoU, the parties will exchange scientific and clinical knowledge and expertise in CNS oncology and in the development and translation of therapeutics for rare, pediatric and high-unmet-need conditions; exchange expertise on clinical development methodologies, including trial design, endpoint selection and response assessment criteria, in support of strengthening clinical development capability in the Emirate; and cooperate on the generation and exchange of real-world evidence and clinical insights relating to CNS tumors in Abu Dhabi, including epidemiological and outcomes data, in accordance with applicable data protection legislation and subject to the required approvals.

The parties will also collaborate on training, capacity-building and professional development initiatives for investigators, clinical research coordinators and allied healthcare professionals, including in clinical trial conduct, neuro-oncology imaging assessment methodologies and molecular diagnostics relevant to CNS tumors, and on the development of Abu Dhabi’s life sciences ecosystem.

The MoU further provides for cooperation on the advancement of clinical research in CNS oncology, including studies of NeOnc’s investigational programs, subject to a separate agreement between the parties and to all applicable regulatory requirements and approvals. Subject to a separate agreement and to all applicable laws, regulations and approvals, the parties will also explore the potential establishment of a regional presence and manufacturing, supply or distribution activities by NeOnc in the Emirate, and the positioning of Abu Dhabi as a potential regional platform for NeOnc’s future development activities. These potential activities remain subject to further assessment, applicable approvals and any necessary definitive agreements.

The MoU is not a binding contract on either party. It takes effect upon signature by representatives of both parties and remains in effect for two years, unless either party gives at least three months’ notice of its wish to terminate. The MoU does not restrict either party from entering into similar arrangements with other institutions.  Continued…  Read this full release and additional news for NTHI by visiting:  https://neonc.com/press-releases/

Other Leading companies advancing next-generation CNS cancer therapies and oncology research:

Neurocrine Biosciences, Inc. (Nasdaq: NBIX) recently announced the initiation of its Phase 2 clinical study of investigational compound NBI-1117567 (NBI-'567) in adults with mild to moderate Alzheimer's disease. NBI-'567 is an orally administered, M1/M4 (M1 preferring) selective muscarinic agonist being evaluated as an adjunctive treatment for cognitive impairment in Alzheimer's disease.

NBI-'567 is designed to directly activate M1 and M4 muscarinic receptors, which are involved in neuronal circuits associated with learning and memory. The study will evaluate the potential of NBI-'567 to improve cognitive performance in adults with mild to moderate Alzheimer's disease who are receiving stable treatment with an acetylcholinesterase inhibitor.

"As a defining characteristic of Alzheimer's disease, cognitive impairment affects memory, attention, and other thinking abilities that significantly impact daily functioning," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "The advancement of NBI-'567 reflects our commitment to delivering new treatment options for this vulnerable population, in addition to our broader strategy to explore the potential of selectively targeting muscarinic receptors across a range of neurological and psychiatric conditions."

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) recently announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. Patent Application No. 17/590,496, titled “Methods and Compositions for Treatment of Rett Syndrome.” The Notice of Allowance indicates that the USPTO has determined that the application meets the requirements for patentability, and the patent is expected to issue following completion of standard administrative steps.

The patent application covers weight-banded dosing of trofinetide for the treatment of Rett syndrome. Following issuance and including six months of pediatric exclusivity, the patent is expected to provide protection for trofinetide in the United States through March 28, 2041, extending patent protections by almost five years. Trofinetide is also protected by a method of use patent for the treatment of Rett syndrome, which expires in July 2036.

Anavex Life Sciences Corp. (NASDAQ: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for central nervous system diseases with significant unmet medical needs, recently announced that it has received notification from the U.S. Food and Drug Administration (FDA) that the Company may proceed with its planned clinical study under its Investigational New Drug Application (IND) for ANAVEX®2-73 (blarcamesine) for Fragile X syndrome (FXS), a genetic condition that is the most common inherited cause of intellectual disability and developmental delay and the most commonly known single-gene cause of autism, for which no FDA-approved treatment currently exists. The IND is now open, enabling clinical investigation of blarcamesine in FXS in the United States. Blarcamesine has previously received Orphan Drug Designation from the FDA for the treatment of Fragile X syndrome.

“FDA clearance of the Fragile X IND is an important milestone that gives Anavex open INDs in all three of our core indications: Alzheimer’s Disease, Rett syndrome, and Fragile X syndrome, and supports our continued momentum of working closely with the FDA as we move our programs forward,” stated Terrie Kellmeyer, Ph.D., Interim Chief Executive Officer. “With an open IND, we are positioned to advance ANAVEX2-73-FXS-001, a planned Phase 2a randomized, double-blind, placebo-controlled, dose-ranging study of the safety, tolerability, and pharmacokinetics of blarcamesine oral solution in approximately 60 adult males with FXS, with behavioral (Aberrant Behavior Checklist revised for FXS “ABCFX”) and cognitive (NIH Toolbox) measures included as secondary endpoints. We believe blarcamesine’s differentiated mechanism of action, including its targeting of sigma-1 receptors, uniquely positions Anavex with the potential to pioneer a transformative treatment approach for patients and families affected by Fragile X syndrome. Newly published peer-reviewed data in Frontiers in Pharmacology further strengthen the scientific rationale for this program.”

Merck (NYSE: MRK), known as MSD outside of the United States and Canada, recently announced that first-time results from three pivotal clinical trials will be presented during late-breaking Presidential Symposia at the European Society for Medical Oncology (ESMO) Congress 2026 in Madrid, Spain from Oct. 23-27. Additionally, more than 90 additional abstracts across more than 20 tumor types from the company’s pipeline and portfolio will be presented throughout the meeting.

“New data at ESMO, including three Presidential Symposia from three pivotal trials, reflect the strength and depth of Merck’s industry-leading oncology portfolio and pipeline,” said Dr. Marjorie Green, senior vice president and head of oncology global clinical development, Merck Research Laboratories. "We are proud to share Phase 3 data for the first time from intismeran and sac-TMT, alongside new research from our oncology portfolio, reflecting our commitment to advancing research across a range of cancer types."

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