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NeOnc Technologies Holdings, Inc. Receives Rare Pediatric Disease Designation for NEO100 in Treatment of Pediatric-Type Diffuse High-Grade Gliomas

1. NTHI received FDA's Rare Pediatric Disease Designation for NEO100. 2. NEO100 targets a serious, rare pediatric brain cancer. 3. The designation offers eligibility for a valuable Priority Review Voucher. 4. NTHI aims to advance NEO100 through clinical trials for pediatric gliomas. 5. The company has a robust drug candidate portfolio and strong patent protections.

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Why Bullish?

The FDA's designation could enhance NTHI's market credibility and expedite commercialization, similar to past cases where rare disease designations led to price surges. Historical examples include companies like Amgen benefitting from similar FDA designations, which resulted in significant stock performance improvements post-announcement.

How important is it?

The rarity of the condition and the FDA designation significantly elevate NTHI’s profile in the biotechnology field. Additionally, the potential for transformative therapies in pediatric oncology directly aligns with current medical needs, increasing investor interest.

Why Long Term?

Given the lengthy regulatory review process and NEO100's clinical development timeline, substantial benefits may manifest over the next few years. Ongoing clinical trials can create incremental value as results are released.

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AMGNREGNVRTX
March 31, 2025 08:05 ET  | Source: NeOnc Technologies Holdings, Inc. WESTLAKE VILLAGE, Calif., March 31, 2025 (GLOBE NEWSWIRE) -- NeOnc Technologies Holdings, Inc. (NASDAQ Global Market: NTHI), a clinical-stage medical biotechnology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to perillyl alcohol (NEO100™) for the treatment of pediatric-type diffuse high-grade gliomas, a serious and life-threatening condition affecting children and adolescents. The designation, issued under Section 529(a)(3) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), recognizes the urgent need for treatment options for pediatric-type diffuse high-grade gliomas. The FDA’s decision is based on findings that the disease primarily affects individuals from birth to 18 years and meets the statutory definition of a “rare disease or condition.” “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, 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.” “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,” said Dr. Thomas Chen, CEO and Chief Science Officer of NeOnc Technologies Holdings, Inc. “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.” 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. The PRV program is designed to encourage the development of new drugs and biologics for rare pediatric diseases by providing companies with an expedited regulatory review process. These vouchers, which are transferable and have been sold in recent years, have significant strategic and financial value, depending on market conditions and demand. “Currently, there is a dynamic market for PRV’s, which are quite valuable,” added Heshmatpour. “This potential asset further underscores the value of our progress, especially for the patients and families we aim to serve.” As NeOnc Technologies continues to progress NEO100 through clinical trials, the company remains dedicated to working closely with the FDA and the broader medical community to bring this promising therapy to children in need. The company encourages collaboration with researchers, healthcare providers, and patient advocacy groups to further accelerate advancements in pediatric brain cancer treatment. ABOUT NEONC TECHNOLOGIES HOLDINGS, INC.NeOnc Technologies Holdings, Inc. is a clinical-stage life sciences company focused on the development and commercialization of central nervous system therapeutics that are designed to address the persistent 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. These proprietary chemotherapy agents have demonstrated positive effects in laboratory tests on various types of cancers and in clinical trials treating malignant gliomas. NeOnc’s NEO100™ and NEO212™ therapeutics are in Phase II human clinical trials and are advancing under FDA Fast-Track and Investigational New Drug (IND) status. The company has exclusively licensed an extensive worldwide patent portfolio from the University of Southern California consisting of issued patents and pending applications related to NEO100, NEO212, and other products from the NeOnc patent family for multiple uses, including oncological and neurological conditions. For more about NeOnc and its pioneering technology, visit neonctech.com. Important Cautions Regarding Forward Looking Statements All statements other than statements of historical facts included in this press release are "forward-looking statements" (as defined in the Private Securities Litigation Reform Act of 1995). Generally, such forward-looking statements include statements regarding expectations, possible or assumed future actions, business strategies, events or results of operations, including statements regarding expectations or predictions or future financial or business performance or conditions and those statements that use forward-looking words such as "projected," "expect," "possibility" and "anticipate," or similar expressions. The achievement or success of the matters covered by such forward-looking statements involve significant risks, uncertainties, and assumptions. Actual results could differ materially from current projections or implied results. The Company cautions that statements and assumptions made in this news release constitute forward-looking statements and make no guarantee of future performance. Forward-looking statements are based on estimates and opinions of management at the time statements are made. The information set forth herein speaks only as of the date hereof. The Company and its management are under no obligation, and expressly disclaim any obligation, to update, alter or otherwise revise any forward-looking statements following the date of this news release, whether because of new information, future events or otherwise, except as required by law. “NEO100” is a registered trademark of NeOnc Technologies Holdings, Inc. Company Contact: Patrick WaltersChief Operations OfficerNeOnc Technologies Holdings, Inc. info@neonc.com Investor Relations: Roger Pondel / Laurie BermanPondelWilkinson Inc.(310) 279-5980rpondel@pondel.comlberman@pondel.com

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