NeOnc Technologies Holdings, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to their investigational treatment NEO100 (perillyl alcohol) for pediatric-type diffuse high-grade gliomas, an aggressive and life-threatening form of brain cancer affecting children and adolescents.
The designation, issued under Section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act, acknowledges that the disease primarily affects individuals from birth to 18 years of age and qualifies as a rare disease. This regulatory milestone could accelerate the development pathway for this novel therapeutic approach.
Significance of the Designation
The Rare Pediatric Disease Designation makes NeOnc Technologies eligible to receive a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval of NEO100's marketing application. These vouchers, which can be used to expedite FDA review of future drug applications or sold to other companies, have significant strategic and financial value in the pharmaceutical industry.
"This designation marks a significant milestone in our efforts to develop innovative therapies for children battling this aggressive form of brain cancer," said Amir Heshmatpour, Executive Chairman of NeOnc Technologies Holdings, Inc. "We remain committed to advancing NEO100 through clinical development to bring new hope to patients and families facing this devastating disease."
Heshmatpour also noted the potential financial implications, stating, "Currently, there is a dynamic market for PRVs, which are quite valuable. This potential asset further underscores the value of our progress, especially for the patients and families we aim to serve."
Novel Treatment Approach
NEO100 represents an innovative approach to treating brain tumors, as it is administered intranasally rather than through traditional routes like injection or oral medication. This delivery method may allow the drug to reach the brain more efficiently while potentially reducing systemic side effects.
Dr. Thomas Chen, CEO and Chief Science Officer of NeOnc Technologies Holdings, Inc., emphasized the importance of this development: "Receiving the Rare Pediatric Disease Designation for NEO100 is a crucial step forward in our mission to develop effective treatments for children facing diffuse high-grade gliomas. This designation not only validates the potential of our research but also strengthens our commitment to delivering innovative therapies that can make a real difference in children's lives."
Clinical Development Plans
A Phase 1b clinical trial is being prepared to evaluate NEO100 in pediatric patients with diffuse high-grade gliomas. The study will assess safety, effectiveness, and determine the optimal dosage through a controlled dose-escalation approach.
The trial will be open to patients with several types of aggressive pediatric brain tumors, including:
- Newly diagnosed and recurrent diffuse midline glioma
- Diffuse hemispheric glioma
- High-grade gliomas
- Other malignant brainstem or posterior fossa tumors
Addressing an Unmet Medical Need
Pediatric-type diffuse high-grade gliomas represent one of the most challenging forms of childhood cancer, with limited treatment options and poor prognosis. Current standard treatments often include surgery (when possible), radiation therapy, and chemotherapy, but outcomes remain unsatisfactory for many patients.
The intranasal delivery system being developed for NEO100 builds on previous research suggesting this route may enhance drug delivery to the brain while minimizing systemic exposure. This approach could be particularly valuable in pediatric patients, where minimizing treatment-related toxicity is especially important.
About NeOnc Technologies
NeOnc Technologies Holdings, Inc. (NASDAQ: NTHI) is a clinical-stage medical biotechnology company focused on developing central nervous system therapeutics designed to overcome the blood-brain barrier. The company's NEO drug development platform has produced a portfolio of novel drug candidates with patent protections extending to 2038.
In addition to NEO100, the company is advancing NEO212, another candidate in Phase II human clinical trials under FDA Fast-Track and Investigational New Drug status. Both therapeutics have demonstrated positive effects in laboratory tests on various cancer types and in clinical trials treating malignant gliomas.
The company has licensed an extensive worldwide patent portfolio from the University of Southern California related to NEO100, NEO212, and other products for multiple uses, including oncological and neurological conditions.
As NeOnc Technologies continues advancing NEO100 through clinical development, the company remains committed to working closely with regulatory agencies, the medical community, and patient advocacy groups to bring new hope to children battling these devastating brain cancers.
