FDA Grants Orphan Drug Designation to Ultimovacs' UV1 Vaccine for Mesothelioma Treatment
Monday, October 09, 2023
Ultimovacs ASA (OSE ULTI), a prominent biotechnology company specializing in innovative cancer vaccines, has secured Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for their therapeutic cancer vaccine UV1, intended for the treatment of mesothelioma patients. This designation has been granted based on preliminary data from the Phase II clinical trial named NIPU.
Mesothelioma is a rare and aggressive cancer type characterized by high mortality rates and limited treatment options. Typically, patients with mesothelioma have a history of asbestos exposure, either occupationally or environmentally, and it often takes decades for this specific cancer to manifest.
The Phase II clinical trial NIPU is evaluating the impact of UV1 vaccination in patients with malignant pleural mesothelioma. In this study, UV1 is administered in combination with checkpoint inhibitors ipilimumab and nivolumab and compared to the administration of ipilimumab and nivolumab alone as a second-line treatment following initial platinum-based chemotherapy. The trial, which involved 118 patients and was conducted across multiple countries, including Australia, Denmark, Norway, Spain, and Sweden, commenced in June 2020 and concluded patient enrollment in January 2023. Oslo University Hospital is sponsoring the NIPU study, with support from Bristol-Myers Squibb and Ultimovacs.
The results of this study will be presented at the ESMO Congress in Madrid, scheduled for October 20-24. The presentation will be delivered by the Principal Investigator, Dr. Åslaug Helland, who is a Professor at Oslo University Hospital. The presentation's title is "LBA99 - First survival data from the NIPU trial; A randomized, open-label, phase II study evaluating nivolumab and ipilimumab combined with UV1 vaccination as second-line treatment in patients with malignant mesothelioma."
Carlos de Sousa, the CEO of Ultimovacs, expressed the significance of obtaining FDA orphan drug designation for UV1 in the context of mesothelioma. He also highlighted the urgent need for new therapeutic options for this patient population and emphasized the company's commitment to advancing UV1 for cancer patients.
Orphan drug designation from the FDA is granted to facilitate the development of medicines for rare disorders affecting fewer than 200,000 individuals in the United States. This designation provides certain advantages, including potential market exclusivity for up to seven years upon regulatory approval, exemption from FDA application fees, and tax credits for qualified clinical trials.
UV1 is a therapeutic cancer vaccine designed to stimulate an immune response against the human telomerase (hTERT) enzyme, a crucial component for the proliferation of cancer cells. Telomerase is found in 85-90% of all cancers across various stages of the disease.
Ultimovacs is conducting an extensive clinical development program for the universal cancer vaccine UV1, encompassing various cancer types with distinct characteristics and disease stages. The NIPU trial's top-line data represents the first results among five ongoing randomized Phase II trials within the UV1 program. In addition to malignant pleural mesothelioma, Phase II studies are ongoing for malignant melanoma, head and neck cancer, ovarian cancer, and non-small cell lung cancer. Results from the malignant melanoma and head and neck cancer trials are also expected within the next year. UV1 is a proprietary, patented technology owned by Ultimovacs.
UV1 is a cancer vaccine with a universal design aimed at eliciting a specific T-cell response against telomerase, a protein associated with cancer cells. UV1 is composed of synthetic peptides representing a segment of the telomerase subunit known as reverse transcriptase (hTERT). These peptides have been proven to activate CD4+ T-cells, which are essential for triggering a robust anti-tumor immune response. When UV1 is administered through intradermal injections, antigen-presenting cells (APCs) in the skin encounter the vaccine peptides. These APCs process the peptides and present vaccine epitopes on Human Leukocyte Antigen (HLA) molecules to naive T-cells located in the lymph nodes. These activated, vaccine-specific T-cells then circulate throughout the body, actively searching for cells that display their specific antigen in conjunction with HLA molecules.
Crucially, the UV1 peptides encompass various epitopes that can be presented by a wide range of HLA alleles. This eliminates the need for HLA pre-screening of patients, making the vaccine potentially suitable for a broad population. UV1 is typically administered over three months through eight intradermal injections, often in combination with the immune-modulator GM-CSF.
