Therapyx receives second FDA Orphan Drug Designation for PCX12, a first-in-class immune therapy, for the treatment of malignant glioma
Buffalo New York, September 29, 2025 /PRNewswire/ — Therapyx today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted the company’s request for an orphan drug designation of PCX12, the company’s investigational therapy in development for the treatment of solid tumors. PCX12 has demonstrated compelling efficacy in preclinical models of glioblastoma, and has consequently received an orphan designation for the broader indication of “treatment of malignant glioma.”
Gliomas are the most common primary brain tumor, arising from glial cells in the brain or spinal cord. They range from slow-growing (low-grade) to fast-growing (high-grade/malignant). Symptoms include headaches, seizures, vomiting, personality changes, and fatigue. There are few effective treatments. PCX12 aims to change this.
PCX12 consists of the key immune regulatory cytokine interleukin 12, formulated for intratumoral delivery and sustained release. Its putative immune mechanism of action includes changes in the tumor microenvironment that unmask “immunologically cold” (i.e., largely invisible to the immune system) tumors, enhance incipient or dormant anti-tumor immune responses. Local tumors, as well as distant metastases, are attacked and killed by the resurgent immune response, especially when combined with radiation.
“We are delighted that the FDA has granted an additional orphan drug designation to PCX12 for this rare but deadly tumor,” said Dr. Nejat K. Egilmez, CEO at Therapyx. “Glioblastoma is an immunologically cold tumor and a devastating diagnosis without a cure and with few treatment options. We look forward to advancing PCX12 to the clinic and are currently exploring partnership opportunities to help bring this novel treatment to patients and their families as quickly and safely as possible.”
Therapyx continues the development of its other products, FAPXILTM, GneX12TM and NGoXIMTM towards the clinic. The company also has a deep pipeline of discovery stage products. Moreover, certain products are likely to be eligible for FDA orphan drug designation, as well as 505(b)(2) accelerated regulatory development pathway. Therapyx has developed a proprietary drug delivery system called ExStaM, based on mild encapsulation of highly potent protein therapeutics into nano/microparticles, that preserves bioactivity, achieves controlled tissue specific release, reduced toxicity and shelf-stability. Its lead products are aimed at cancer, mucosal infections and immune mediated inflammatory diseases. For more information on Therapyx, Inc. visit the Company’s website at therapyxinc.com
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