perillyl alcohol (NEO100) / NeOnc Technologies - LARVOL DELTA

A Study of the Safety, Dosing, and Delivery of NEO100 in Patients With Pediatric Brain Tumors (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Neonc Technologies, Inc. | Trial completion date: Oct 2025 ➔ Oct 2026 | Trial primary completion date: Oct 2025 ➔ Oct 2026

Trial completion date • Trial primary completion date • Brain Cancer • CNS Tumor • Diffuse Midline Glioma • Glioma • High Grade Glioma • Oncology • Pediatrics • Solid Tumor

Intranasal novel compound NEO100-facilitated steroid delivery in managing peritumoral edema in rat glioma animal model (SNO 2025) - " The pharmacokinetic (pK) study was performed to compare the efficacy of oral and IN Dexamethasone (Dex) with and without NEO100. IN NEO100-mediated Dex delivery shows great promise compared to oral administration and is also effective in managing peritumoral edema. Other effects on animal survival, blood sugar levels, liver function, metabolite rates, as well as T3/T4 levels, are under investigation."

Preclinical • Astrocytoma • Brain Cancer • CNS Disorders • Cushing’s Disease • Endocrine Disorders • Gastrointestinal Disorder • Glioma • Oncology • Solid Tumor • IDH1

NEO100 facilitates delivery of blood-brain barrier impenetrable drugs (SNO 2025) - "We have shown that NEO100 significantly enhances the brain tumor specific uptake and retention of doxorubicin, dexamethasone, and remdesivir when administered via an intra-nasal route in a formulation that contains NEO100 at concentrations as low as 1%, when compared to traditional oral or IV routes of administration. Understanding how NEO100 functions in manipulating the barrier's physiology is crucial for developing next-generation therapeutics capable of treating CNS diseases effectively. Continued innovation in drug design and delivery technologies is essential for expanding the range of treatable neurological conditions"

Brain Cancer • CNS Disorders • Glioma • Solid Tumor

Intranasal novel compound NEO100-facilitated steroid delivery in managing peritumoral edema in rat glioma animal model (WFNOS 2025) - " The pharmacokinetic (pK) study was performed to compare the efficacy of oral and IN Dexamethasone (Dex) with and without NEO100. IN NEO100-mediated Dex delivery shows great promise compared to oral administration and is also effective in managing peritumoral edema. Other effects on animal survival, blood sugar levels, liver function, metabolite rates, as well as T3/T4 levels, are under investigation."

Preclinical • Astrocytoma • Brain Cancer • CNS Disorders • Cushing’s Disease • Endocrine Disorders • Gastrointestinal Disorder • Glioma • Solid Tumor • IDH1

A Validated UPLC-MS/MS Method for Monitoring Temozolomide and Perillyl Alcohol Metabolites in Biological Matrices: Insights From a Preclinical Pharmacokinetic Study. (PubMed, Biomed Chromatogr) - "PA was mainly distributed to liver and kidneys, with limited brain penetration. The validated method enables preclinical pharmacokinetic and tissue distribution studies, offering valuable insights into TMZ and POH behavior for GBM treatment."

Journal • PK/PD data • Preclinical • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

NeOnc Technologies Holdings, Inc…announced the completion of full patient enrollment in its ongoing NEO100-1 Phase 2a clinical trial. (GlobeNewswire) - "The Company anticipates reporting preliminary data from this fully enrolled cohort in approximately six months, during the second quarter of 2026."

Enrollment closed • P2a data • Glioma

NEO100 facilitates delivery of blood-brain barrier impenetrable drugs (WFNOS 2025) - "We have shown that NEO100 significantly enhances the brain tumor specific uptake and retention of doxorubicin, dexamethasone, and remdesivir when administered via an intra-nasal route in a formulation that contains NEO100 at concentrations as low as 1%, when compared to traditional oral or IV routes of administration. Understanding how NEO100 functions in manipulating the barrier's physiology is crucial for developing next-generation therapeutics capable of treating CNS diseases effectively. Continued innovation in drug design and delivery technologies is essential for expanding the range of treatable neurological conditions"

Brain Cancer • CNS Disorders • Solid Tumor

NeOnc Technologies…to Host Investor Conference Call to Present Data Updates from Ongoing NEO100-1 Phase 1/2a Clinical Trial and Compassionate Use Program (GlobeNewswire) - "During the call, members...will present data updates from the Company’s ongoing Phase 1/2a NEO100-1 clinical trial and compassionate use program, evaluating intranasal delivery of NEO100 (high-purity perillyl alcohol) in patients with recurrent high grade malignant glioma. The discussion will highlight MRI-based radiographic response data, progression-free survival (PFS), and overall survival (OS) trends observed to date, providing important insights into long-term clinical outcomes for patients treated with NEO100."

Clinical • P1/2 data • Glioma

Safety and Efficacy Study in Recurrent or Progressive Grade III or IV IDH1 Mutated Glioma (clinicaltrials.gov) - P1/2 | N=49 | Recruiting | Sponsor: Neonc Technologies, Inc. | Trial completion date: Dec 2024 ➔ Dec 2025 | Trial primary completion date: Dec 2024 ➔ Oct 2025

Trial completion date • Trial primary completion date • Brain Cancer • Glioblastoma • Glioma • Oncology • Solid Tumor • IDH1

Intranasal Terpene Treatment for Glioblastoma: the Neuro-Oncological Potential of Perillyl Alcohol. (PubMed, Neurochem Res) - "This review aims to evaluate the potential of intranasal terpene treatment (ITT) as a novel and non-invasive strategy to bypass the BBB and improve glioblastoma treatment outcomes. A review of recent preclinical and clinical studies on intranasally administered compounds (especially terpenes such as Perillyl alcohol (POH)) is presented in terms of their molecular mechanisms, bioavailability, and clinical effects in the central nervous system (CNS)."

Journal • Review • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Potent Therapeutic Activity of NEO212 in Preclinical Models of Human and Canine Leukaemia and Lymphoma. (PubMed, Vet Comp Oncol) - "We are developing a novel anticancer agent, NEO212, a conjugate of two cancer drugs, the alkylating agent temozolomide (TMZ) and the monoterpene perillyl alcohol (POH). At the same time, NEO212 is well tolerated in dogs at dosages higher than those that achieved therapeutic activity in mouse models. Our study introduces NEO212 as a novel oral cancer drug candidate for both human and veterinary oncology applications."

Journal • Preclinical • Head and Neck Cancer • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Oncology • Oral Cancer • Solid Tumor

Optimization of Eudragit RS100 Nanocapsule Formulation for Encapsulating Perillyl Alcohol and Temozolomide Using Design of Experiments. (PubMed, ACS Nanosci Au) - "Encapsulation efficiencies were 3.7% for POH and 28.5% for TMZ. This nanoencapsulated delivery system offers a promising approach for glioblastoma treatment, potentially enhancing clinical outcomes and reducing treatment-associated toxicity."

Journal • Brain Cancer • CNS Tumor • Glioblastoma • Oncology • Solid Tumor

NeOnc Technologies Holdings, Inc. Expects Full Enrollment in Promising Phase 2a Trial of NEO100-01 for Malignant Gliomas in September (GlobeNewswire) - "NeOnc Technologies Holdings...announced that it expects full enrollment in its Phase 2a clinical trial for its lead therapeutic candidate, NEO100-01 in September. Only six patients remain to complete the trial’s 25-patient enrollment target. This landmark trial, for which read-out data is expected in early 2026, investigates NEO100, a proprietary, highly purified version of perillyl alcohol, delivered intranasally, a non-invasive method designed to bypass the blood-brain barrier through the olfactory and trigeminal nerves."

P1/2 data • Trial status • Glioma

FDA Gives RPDD to NEO100 for Pediatric-Type Diffuse High-Grade Glioma (OncLive) - "The FDA has granted rare pediatric disease designation (RPDD) to perillyl alcohol (NEO100) for the treatment of patients with pediatric-type diffuse high-grade gliomas (HGG)....A phase 1b dose-finding trial...which has yet to begin recruiting patients, will assess NEO100’s safety, brain tumor delivery, and pharmacokinetics in patients with pediatric-type diffuse HGG."

FDA event • Trial status • Glioma

NeOnc Technologies Expands Global Clinical Trial Network with CBCC Global Research to Accelerate Study of Brain Cancer Treatments (GlobeNewswire) - "NeOnc Technologies Holdings, Inc...announced a strategic partnership with CBCC Global Research (CBCC), a premier full-service clinical research organization (CRO). This collaboration will expand NeOnc’s clinical trial capabilities in India, facilitating the advancement of its development-stage neuro-oncology treatment....CBCC will initially recruit for the NEO100-01 Glioblastoma (GBM) clinical trial, which is a Phase 2a study evaluating the survival impact and tolerance of intranasal NEO100, an ultra-purified perillyl alcohol with a focus on the IDH1 mutation component in Grade 3 and 4 GBM tumors....'We are focused on completing our Phase 2 NEO100-01 enrollment this year, with a readout expected 6 to 8 months after that'."

Commercial • P2a data • Glioblastoma

Therapeutic Delivery of circDYM by Perillyl Alcohol Nanoemulsion Alleviates LPS-Induced Depressive-Like Behaviors. (PubMed, Adv Sci (Weinh)) - "This effect is achieved by reducing the CD11b+CD45dim microglia population and iNOS expression, restoring the expression of protein-95 (PSD-95) and synaptophysin. These findings indicated that PANE represents an efficient platform for delivering circRNAs to the brain, and intranasal administration of PANE2-4-circDYM is a promising strategy for ameliorating LPS-induced depressive-like behaviors."

Journal • CNS Disorders • Depression • Genetic Disorders • Infectious Disease • Mental Retardation • Oncology • Psychiatry • ITGAM • SYP

Optimized mucus adhesion and penetration of lipid-polymer nanoparticles enables effective nose-to-brain delivery of perillyl alcohol for glioblastoma therapy. (PubMed, Drug Deliv Transl Res) - "In vitro assays demonstrated that LPNP-POH increased the cytotoxicity and reduced the tumor growth of U87MG glioma cells. These results highlighted that the engineered formulation, with optimized mucoadhesiveness and mucus penetration properties, improved nose-to-brain delivery of POH, offering a promising potential for glioblastoma therapy."

Journal • Brain Cancer • CNS Disorders • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor

Patient with recurrent grade 4 astrocytoma responding favorably to intranasal delivery of NEO100, highly pure perillyl alcohol: illustrative case. (PubMed, J Neurosurg Case Lessons) - "The authors present a case in which IN cancer therapy with NEO100 was well tolerated and was associated with striking tumor regression, providing further evidence that this novel conceptual approach to cancer therapy might become useful for the improved treatment of recurrent glioma. https://thejns.org/doi/10.3171/CASE24683."

Journal • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor • IDH1

Bringing intranasal drug delivery for malignancies in the brain to market. (PubMed, Expert Opin Drug Deliv) - No abstract available

Journal • Brain Cancer • CNS Tumor • Glioma • Meningioma • Oncology • Solid Tumor

Safety and Efficacy Study in Recurrent or Progressive Grade III or IV IDH1 Mutated Glioma (clinicaltrials.gov) - P1/2 | N=49 | Recruiting | Sponsor: Neonc Technologies, Inc. | Trial primary completion date: Jun 2024 ➔ Dec 2024

Trial primary completion date • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Oncology • Solid Tumor • IDH1

A Study of the Safety, Dosing, and Delivery of NEO100 in Patients with Pediatric Brain Tumors (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Neonc Technologies, Inc. | Trial completion date: Oct 2024 ➔ Oct 2025 | Initiation date: May 2024 ➔ Dec 2024 | Trial primary completion date: Oct 2024 ➔ Oct 2025

Trial completion date • Trial initiation date • Trial primary completion date • Brain Cancer • CNS Tumor • Diffuse Midline Glioma • Glioma • Oncology • Pediatrics • Solid Tumor

NeOnc Technologies Begins Enrollment for Phase 2 Clinical Trial of NEO100-02 for Patients Afflicted with Meningioma, Marking Second Concurrent Phase 2 Study of Leading CNS Cancer Drug Candidate (GlobeNewswire) - "NeOnc Technologies Holdings, Inc...has begun patient enrollment for the Phase 2 clinical trial of NEO100-02, the company’s first of two drug candidates proceeding through four concurrent clinical trials for various indications and patient populations. The Phase 2 study of NEO100-02 seeks further safety and preliminary evidence of the biological effects of the novel drug on patients afflicted with residual, progressive or recurrent high-grade meningioma."

Enrollment open • Meningioma

NEO212, temozolomide conjugated to NEO100, exerts superior therapeutic activity over temozolomide in preclinical chemoradiation models of glioblastoma. (PubMed, Neurooncol Adv) - "NEO212 is a candidate drug to potentially replace TMZ within the standard Stupp protocol. It has the potential to become the first chemotherapeutic agent to significantly extend overall survival in TMZ-resistant patients when combined with radiation."

Journal • Preclinical • Brain Cancer • CNS Tumor • Glioblastoma • Hematological Malignancies • Oncology • Solid Tumor

Therapeutic effect of NEO400, perillyl alcohol conjugated to linoleic acid, in a mouse model of UV-induced skin damage. (PubMed, Photochem Photobiol) - "None of these skin-protective effects could be achieved with Aloe vera gel, a popular and widely used post-exposure remedy. Our study suggests that NEO400 holds potential as a regenerative treatment for excessively UVR-exposed skin."

Journal • Preclinical

Molecular pharmacology and therapeutic advances of monoterpene perillyl alcohol. (PubMed, Phytomedicine) - "This review systematically summarizes the recent advances in POH and highlights its therapeutic effects and potential mechanisms as well as the clinical settings, which is helpful to develop POH into functional food and new candidate drug for prevention and management of diseases. Future studies are needed to conduct more biological activity studies of POH and its derivatives, and check their clinical efficacy and potential side effects."

Journal • Review • Asthma • Immunology • Metabolic Disorders • Oncology • BAX • BCL2 • CASP3 • CCND1 • CDKN1A • HIF1A • NLRP3 • NOS3 • PARP1 • STAT3