Eflornithine Hydrochloride

SEATTLE, Oct. 2, 2025 /PRNewswire/ -- Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing novel therapies for rare and difficult-to-treat cancers, today announced 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 patients with neuroblastoma. "Receiving Orphan Drug Designation for AMXT 1501 in combination with DFMO represents an important milestone in our mission to develop innovative therapies for children with life-threatening cancers," said Mark Burns, PhD, Chief Scientific Officer and President of Aminex Therapeutics, Inc. "We are committed to working closely with regulators, investigators and patient advocacy groups, to accelerate the clinical development efforts for AMXT 1501 in combination with DFMO for the treatment of neuroblastoma and other childhood and adult tumor types in collaboration with the Beat Childhood Cancer Research Consortium." The FDA grants ODD to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders. ODD provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to a unique seven years of market exclusivity for the disease in which it has ODD, which is an added value in that it is separate from intellectual property protection. "Neuroblastoma is a rare childhood cancer that unfortunately, accounts for 12-15% of all pediatric cancer deaths in the United States," said Dr. Giselle Sholler, chief of the division of Pediatric Hematology and Oncology at Penn State Health Children's Hospital, director of Pediatric Oncology Research and professor of Pediatrics and Neuroscience at Penn State College of Medicine and founder and chair of the Beat Childhood Cancer Research Consortium. "We believe this combination has the potential to build upon the FDA approval of DFMO to further improve clinical outcomes for children with neuroblastoma and other rare childhood cancers." The Beat Childhood Cancer Research Consortium at Penn State College of Medicine is currently initiating a Phase 1/2 clinical trial in pediatric patients who will be administered AMXT 1501 in combination with DFMO entitled "A Dose Escalation Study Using Eflornithine (DFMO) and AMXT 1501 Followed by a Randomized Controlled Trial of DFMO with or without AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas" (NCT06465199). Aminex Therapeutics, Inc. is currently preparing to initiate clinical trials to further evaluate the safety and efficacy of AMXT 1501 in combination with DFMO in metastatic melanoma and in breast cancer. About Neuroblastoma Neuroblastoma is an aggressive pediatric cancer of the nervous system and the most common extracranial solid tumor in children. Children with high-risk neuroblastoma face survival rates of less than 50% despite intensive multimodal therapy. Furthermore, patients who relapse following conventional standard of care therapies have a long-term survival rate of <10%, underscoring the urgent need for novel treatment approaches. About AMXT 1501 and DFMO AMXT 1501 is a novel polyamine transport inhibitor designed to block the uptake of polyamines, which are essential for tumor growth and survival. In combination with DFMO, an irreversible inhibitor of ornithine decarboxylase, AMXT 1501 is intended to comprehensively suppress polyamine metabolism, a pathway shown to be critical in the development, metastasis and resistance to treatment of neuroblastoma and other cancers. DFMO is an established inhibitor of polyamine biosynthesis. Together, the combination aims to comprehensively inhibit polyamine metabolism and tumor growth. About Aminex Therapeutics, Inc. Aminex Therapeutics, Inc. is a clinical-stage biotechnology company focused on the development of a novel small molecule combination immunotherapy for the treatment of cancer. Aminex has advanced AMXT 1501 through target discovery, patenting, preclinical research and into clinical development. For more information, please visit . About Beat Childhood Cancer Research Consortium The Beat Childhood Cancer Research Consortium is an international academic group of 50+ universities and children's hospitals that offer a worldwide network of childhood cancer clinical trials coordinated through Penn State College of Medicine. They have opened over 27 clinical trials based on the research from collaborating investigators who are linked with laboratory programs developing novel therapies for high-risk pediatric cancers. Their mission is to improve outcomes for children with cancer. About Penn State College of Medicine Located on the campus of Penn State Health Milton S. Hershey Medical Center in Hershey, Pa., Penn State College of Medicine boasts a portfolio of more than $150 million in funded research. Projects range from the development of artificial organs and advanced diagnostics to groundbreaking cancer treatments and understanding the fundamental causes of disease. Enrolling its first students in 1967, the College of Medicine has more than 1,700 students and trainees in medicine, nursing, the health professions and biomedical research on its two campuses. SOURCE Aminex Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

LOUISVILLE, Ky., Aug. 4, 2025 /PRNewswire/ -- US WorldMeds (USWM) reported the successful closing of the previously announced acquisition of Adaptimmune Therapeutics plc's (Adaptimmune) cell–therapy assets—including TECELRA® (afamitresgene autoleucel), lete–cel, afami–cel, and uza–cel. The acquisition was first announced on July 28, 2025 and has now been finalized. Under the terms of the Asset Purchase Agreement, USWM paid $55 million in cash at closing and will fund up to an additional $30 million in performance-based milestone payments tied to commercial and regulatory outcomes. Adaptimmune has retained rights to its pre–clinical programs, including PRAME– and CD70–directed T–cell therapies, and its allogeneic pipeline. Key Highlights: Patient care continuity: TECELRA will remain available to patients without interruption, now under USWM's stewardship. Future development: USWM plans to bring lete–cel to the U.S. market, with potential regulatory approval anticipated in 2026, and will continue development of uza–cel in collaboration with Galapagos. Employee transition: Approximately half of Adaptimmune's U.S.-based workforce is being offered roles at USWM to support commercialization, production, and ongoing development of the acquired assets. Transition support: Adaptimmune is providing transition services through June 2026 to ensure operational continuity. Breck Jones, Chief Executive Officer of US WorldMeds, commented: "With the transaction now complete, we are excited to officially welcome Adaptimmune's programs and people into our organization. We are committed to building on the strong foundation Adaptimmune has established and advancing these therapies to bring lasting impact to patients with high unmet needs." TD Cowen acted as financial advisor, and Ropes & Gray LLP provided legal counsel, to Adaptimmune. Gibson, Dunn & Crutcher LLP provided legal counsel to US WorldMeds. The transaction was financed by debt financing led by funds managed by Oaktree Capital Management, L.P. ("Oaktree"), with participation from funds managed by Athyrium Capital Management, LP ("Athyrium"). Indication TECELRA is a medicine, called a genetically modified autologous T cell immunotherapy, that is used to treat synovial sarcoma. It is used when other kinds of treatment do not work. TECELRA is different from other cancer medicines because it is made from your own white blood cells that are made to recognize and attack your cancer cells. Your healthcare provider will perform tests to see if TECELRA is right for you. TECELRA is approved based on patient response data. Additional data are needed to confirm the clinical benefit of TECELRA. It is not known if TECELRA is safe and effective in children. Important Safety Information Important Warning: You will likely be in a hospital before and after getting TECELRA. TECELRA may cause side effects that can be severe or life-threatening. Call your healthcare provider or get emergency help right away if you get any of the following: fever (100.4°F/38°C or higher); chills/shivering; difficulty breathing; fast or irregular heartbeat; low blood pressure; fatigue; severe nausea, vomiting, or diarrhea; severe headache; or new skin rash. Tell all your healthcare providers that you were treated with TECELRA. After getting TECELRA, you will be monitored daily at the healthcare facility for at least 7 days after the infusion. You should plan to stay close to a healthcare facility for at least 4 weeks. Do not drive, operate heavy machinery, or do other activities that could be dangerous for at least 4 weeks after you get TECELRA. Your healthcare provider will do blood tests to follow your progress. It is important that you have your blood tested. If you miss a scheduled appointment for your collection of blood, call your healthcare provider as soon as possible to reschedule. Before you receive TECELRA, tell your healthcare provider about all the medicines and supplements you take and your medical conditions, including: seizure, stroke, confusion, or memory loss; heart, liver, or kidney problems; low blood pressure; lung or breathing problems; recent or active infection; past infections that can be reactivated following treatment with TECELRA; low blood counts; pregnancy, you think you may be pregnant, or plan to become pregnant; breastfeeding; or taking a blood thinner. The most common side effects of TECELRA include nausea, vomiting, fatigue, infection, constipation, fever (100.4°F/38°C or higher), abdominal pain, difficulty breathing, decreased appetite, diarrhea, low blood pressure, back pain, fast heart rate, chest pain, general body swelling, low white blood cells, low red blood cells, and low platelets. You are encouraged to report side effects to the FDA at (800) FDA–1088 or . Please see Full Prescribing Information, including Medication Guide, at Tecelra.com. About US WorldMeds US WorldMeds is a U.S.-based pharmaceutical company focused on rare and specialty treatments. Our portfolio includes approved therapies for rare diseases and bleeding disorders, including approved therapies Iwilfin® an Revonto®. With this acquisition, we expand further into oncology and cell therapy, reinforcing our commitment to bringing life-changing therapies to patients. Media Contact: Name: Jessica Tracy Title: VP, Commercial Operations US WorldMeds Ventures, LLC Phone: 1-833-935-6676 Email: [email protected] SOURCE US WorldMeds WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED