On March 12, 2026 Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing a novel metabolic-targeted therapy to treat adult and pediatric cancers, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG). This is the company’s second Orphan Drug Designation, following the ODD granted in October 2025 for neuroblastoma.
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"This second Orphan Drug Designation is a powerful validation of AMXT 1501’s potential to make a meaningful difference for patients facing some of the most devastating cancers," said Mark Burns, PhD, Chief Scientific Officer and President of Aminex Therapeutics. "DIPG is heartbreaking — children diagnosed with this brain tumor have virtually no effective treatment options and very little time. We are committed to changing that."
DIPG is an aggressive brainstem tumor that primarily strikes children aged 5 to 10, with a median survival of less than 12 months from diagnosis. There is currently no cure.
For Families of Children with DIPG and Other High-Risk Cancers: A Trial Is Now Enrolling
The Beat Childhood Cancer Research Consortium at Penn State College of Medicine, in partnership with Aminex, is actively enrolling patients in a national Phase 1/2 clinical trial of AMXT 1501 plus DFMO in pediatric patients with DIPG, neuroblastoma, sarcomas and other high-risk childhood cancers. The trial is planned to open at 50 clinics nationwide and enroll patients. For more information, visit ClinicalTrials.gov (NCT06465199) to learn more.
AMXT 1501 is also being evaluated in adults with solid tumors including breast cancer and metastatic melanoma in an active Phase 1b/2 multicenter trial (NCT07287917.)
The FDA’s Orphan Drug Designation provides Aminex with development incentives including tax credits, fee waivers, and seven years of market exclusivity upon potential approval.
(Press release, Aminex Therapeutics, MAR 12, 2026, View Source;a-highly-aggressive-childhood-brain-cancer-302711779.html [SID1234663514])