Therapyx receives FDA Orphan Drug Designation for PCX12, a first-in-class immune therapy, for the treatment of pancreatic cancer
Buffalo New York, June 10, 2024 /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 remarkable efficacy in preclinical models of pancreatic cancer, against which the drug will be initially deployed in the clinic, and for which the orphan drug designation has been granted.
Pancreatic cancer is a highly aggressive disease occurring when cells in the pancreas develop mutations, causing rapid, uncontrolled growth, usually starting in the ducts (adenocarcinoma). It is difficult to detect early, often showing no symptoms until advanced, with common signs including jaundice, upper abdominal pain, and unexplained weight loss.
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 orphan drug designation to PCX12 in this rare but deadly disease. Combined with our recently awarded patent for PCX12 in combination with radiation, this highlights the importance of advancing new treatments for patients living with these dreaded solid tumors,” said Dr. Nejat K. Egilmez, CEO at Therapyx. “Pancreatic cancer is an immunologically cold tumor and 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
CAUTIONARY STATEMENT: This press release may contain forward-looking statements concerning the potential and prospects of the Company’s drug discovery program and its drug candidates. Any statement describing a goal, expectation, intention or belief of the Company is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including the ability to complete preclinical and clinical trials successfully and within specified timelines, if at all; the ability to obtain regulatory approval; the Company’s future capital needs and ability to obtain additional funding; the ability of the Company to protect its intellectual property rights and to not infringe the intellectual property rights of others; the development of competitive products by other companies; and other risks detailed from time to time in the Company’s filings and press releases. Actual results may differ materially from those suggested in this press release.
