NeOnc's AI-Powered Breakthrough Amplifies Brain Cancer Drug Efficacy

CALABASAS, CA – December 01, 2025 – In the high-stakes world of oncology, treating brain cancer remains one of the most formidable challenges, largely due to the brain's own defense mechanism: the blood-brain barrier. Today, NeOnc Technologies (Nasdaq: NTHI) announced a significant development that leverages artificial intelligence and focused ultrasound to potentially bypass this hurdle, representing a major stride in a field desperate for innovation.

New preclinical findings, stemming from a collaboration with the University of Southern California (USC), show that NeOnc's lead drug candidate, NEO100, becomes markedly more potent when combined with focused ultrasound. The study, published on the preprint server bioRxiv, suggests this combination creates a powerful sonodynamic therapy capable of destroying a wide range of primary and metastatic brain tumor cells. For a clinical-stage company like NeOnc, this isn't just a scientific curiosity; it's a strategic expansion of its core asset, potentially unlocking new treatment paradigms and significant commercial opportunities.

The Engine of Discovery: AI and Bioprinted Tumors

What makes this announcement particularly compelling is not just the result, but the method. The breakthrough was enabled by a sophisticated, AI-driven platform that sidesteps many of the time-consuming and ethical hurdles of traditional drug discovery. At the heart of this process is a proprietary technology NeOnc calls a New Approach Methodology (NAM), built on patented rapid magnetic 3D bioprinting.

Instead of relying on conventional cell cultures or lengthy animal studies, researchers at USC, led by NeOnc’s Chief Clinical Officer Dr. Josh Neman, can now generate physiologically relevant, patient-derived tumor organoids—miniature, lab-grown versions of a patient's specific cancer—in a matter of hours. This is a dramatic acceleration from the weeks or months typically required. These bioprinted tumor spheroids, which included aggressive cancers like glioblastoma, pediatric medulloblastoma, and metastases from breast and lung cancer, served as the testbed for the study.

Layered on top of this rapid prototyping is a powerful AI neural network. Trained on over 200 molecular descriptors, the AI was tasked with sifting through countless compounds to predict which would be most effective as a sonosensitizer—an agent activated by ultrasound. The platform consistently identified NEO100 as a top candidate, predicting its ability to both penetrate the blood-brain barrier and exhibit potent therapeutic activity when stimulated by sound waves. The subsequent validation on the bioprinted organoids confirmed the AI’s prediction, showing a marked enhancement in tumor-killing when NEO100 was paired with focused ultrasound.

This methodology is rapidly gaining traction with regulators. Both the FDA and NIH have recently issued guidance encouraging the use of NAMs to reduce reliance on animal testing and accelerate the development of new medicines. By building its discovery engine around this forward-looking approach, NeOnc is not only speeding up its internal R&D but also aligning itself with the future of regulatory science.

A New Clinical Horizon for NEO100

The clinical implications of this discovery are profound. NEO100 is already a promising asset, currently in a pivotal Phase II trial for recurrent high-grade gliomas, with Orphan Drug and Fast Track designations from the FDA. The company recently completed enrollment for that trial and expects a preliminary data readout in the second quarter of 2026. The existing data is encouraging, with a previous cohort showing a 21% radiographic response rate and 44% progression-free survival at six months.

However, the addition of focused ultrasound transforms the drug’s potential. Sonodynamic therapy is a non-invasive approach where a benign agent, the sonosensitizer, is administered and then activated precisely at the tumor site using focused ultrasound. This localized activation triggers a cytotoxic effect, killing cancer cells while minimizing damage to surrounding healthy brain tissue. It also temporarily opens the blood-brain barrier, allowing for better drug penetration.

By proving NEO100 is a potent sonosensitizer, NeOnc has effectively opened a second, potentially much larger, front for its lead candidate. The preclinical study demonstrated efficacy across a broad spectrum of tumor types, suggesting a future where a single drug could be adapted for glioblastoma, medulloblastoma, and difficult-to-treat brain metastases. As stated by Amir Heshmatpour, Executive Chairman and President of NTHI, “This breakthrough provides strong external validation for NEO100’s potential as a first-in-class, noninvasive sonodynamic therapeutic. The integration of AI, 3D bioprinting, and ultrasound significantly expands NEO100’s commercial and clinical opportunity beyond current indications.”

Capital, Competition, and Corporate Strategy

For investors and industry analysts, this development repositions NeOnc within the competitive CNS oncology landscape. The global brain tumor therapeutics market is projected to exceed $5 billion by 2030, driven by an urgent need for more effective treatments for diseases like glioblastoma, which currently dominates the market. While giants like Roche, Merck, and Pfizer are key players, the field is ripe for disruption by smaller, more agile biotechs with innovative platforms.

NeOnc’s strategy appears to be one of leveraging a deep well of intellectual property—with patent protections extending to 2038—and cutting-edge technology to de-risk and accelerate its pipeline. While the company is not yet profitable, reporting a net loss of $8.6 million in its most recent quarter, it has secured critical funding, including a $50 million strategic partnership with Quazar Investment and $2.5 million in NIH grants. This capital is crucial as the company moves NEO100 through its current trials and prepares to advance the newly validated ultrasound combination toward human studies.

NTHI's stock has been volatile, reflecting the high-risk, high-reward nature of clinical-stage biotech. It has seen significant gains following positive news but remains well below its all-time high from early 2025. This announcement adds a significant catalyst to the company’s narrative, providing a clear vision for pipeline expansion that complements its ongoing Phase II efforts. The successful marriage of an existing clinical asset with a next-generation delivery and activation method is a powerful story for investors, demonstrating a clear strategy for maximizing the value of its core science. The next critical step will be translating these compelling preclinical results into a concrete clinical trial plan and, ultimately, demonstrating safety and efficacy in patients.