Neonc Technologies Holdings, Inc. (NASDAQ: NTHI) has reported updated clinical results from its ongoing phase 1/2a and compassionate-use experience evaluating intranasal Neo100 in patients with recurrent WHO grade III/IV IDH1-mutant astrocytoma. Since the last announcement, an additional patient has achieved both durable long-term survival and radiographic remission, bringing the total number of patients in the expanded clinical cohort to 25.
The updated results show that treatment with intranasally delivered Neo100 has resulted in significant radiographic remission in 6 of 25 patients, representing a 3x increase over the 8% response rates typically reported with salvage therapies in recurrent high-grade gliomas. Additionally, 44% of patients achieved six-month progression-free survival (PFS-6), exceeding historical benchmarks of 21–31% for IDH1-mutant recurrent high-grade gliomas. Nine of 25 patients (36%) remain alive ≥18 months following initiation of Neo100, providing further evidence of meaningful long-term survival in this heavily pretreated population.
No significant toxicity has been observed with intranasal administration of Neo100, even with prolonged and chronic dosing.
Amir F. Heshmatpour, Executive Chairman, President & CEO of Neonc Technologies Holdings, Inc., emphasized the significance of these results, stating that the addition of another patient achieving both long-term survival and radiographic remission further validates the compelling and differentiated therapeutic signal of Neo100. He also highlighted that radiographic responses are now 300% higher than the rate historically reported with salvage therapies, exceeding expectations.
Dr. Thomas Chen, Founder, Vice-Chairman, and Chief Medical Officer, commented that with each additional durable responder, the data increasingly support the hypothesis that intranasal Neo100 may be a first-in-class, CNS-penetrant metabolic therapy capable of inducing sustained radiographic remission and multi-year survival in recurrent IDH1-mutant gliomas.
The key data highlights include: Radiographic Response: 24% (6 of 25 patients) achieved significant radiographic remission, exceeding the <8% typically observed with salvage therapies for recurrent gliomas. Progression-Free Survival (PFS-6): 44% of patients achieved 6-month progression-free survival, outperforming historical benchmarks of 21–31% for IDH1-mutant recurrent high-grade gliomas. Long Term Survival: 36% (9 of 25) demonstrated durable survival ≥18 months post-initiation of Neo100. Tolerability: No significant toxicity observed even with prolonged, chronic intranasal administration.
These findings indicate that Neo100 could represent a first-in-class, CNS-penetrant metabolic therapy potentially inducing significant radiographic response and extending survival in patients with recurrent grade III/IV IDH1-mutant astrocytoma, offering a major advancement over past approaches predominantly limited to palliative care.
Neonc Technologies Holdings, Inc. is a clinical-stage life sciences company focused on the development and commercialization of central nervous system therapeutics designed to address the challenges in overcoming the blood-brain barrier. The company’s Neo™ drug development platform has produced a portfolio of novel drug candidates and delivery methods with patent protections extending to 2038.
For more information about Neonc and its pioneering technology, visit neonc.com. Following these announcements, the company's shares moved -7.73%, and are now trading at a price of $8.71. For more information, read the company's full 8-K submission here.
