The Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) was granted based on results from the randomized Phase II clinical trial NIPU, which showed improved overall survival with UV1 in combination with ipilimumab and nivolumab in patients with malignant pleural mesothelioma UV1 has also received U.S. Food and Drug Administration ODD for UV1 in the treatment of patients with mesothelioma and malignant stage IIB-stage IV melanoma
Oslo, February 19, 2024: Ultimovacs ASA (“Ultimovacs”) (OSE ULTI), a clinical-stage biotechnology leader in novel immunotherapeutic cancer vaccines, today announced that the European Medicines Agency (EMA) has issued a positive opinion on the company’s application for Orphan Drug Designation (ODD) for its therapeutic cancer vaccine UV1 for the treatment of mesothelioma. The designation was granted based on results from the Phase II clinical trial, NIPU evaluating UV1 added to ipilimumab and nivolumab treatment in patients with malignant pleural mesothelioma. “The EMA’s Orphan Drug Designation for UV1 in mesothelioma is an important step forward in the development of our cancer vaccine in this indication,” said Carlos de Sousa, CEO of Ultimovacs. “It allows for important regulatory and commercial benefits and provides us with the potential to rapidly advance UV1 for a patient population with poor prognosis and a high unmet medical need.” To qualify for orphan designation in the EU, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition affecting fewer than five in 10,000 people in the EU, based on data that support that the investigational medicine may produce clinically significant benefits over existing treatments. ODD provides companies with certain benefits and incentives, including ten years of market exclusivity upon reaching the market, clinical protocol assistance, access to a centralized marketing authorization procedure, and reduced regulatory fees. Mesothelioma is a rare and aggressive form of cancer with a high mortality rate and few therapeutic options. Patients with mesothelioma commonly have a history of occupational or environmental exposure to asbestos, and it typically takes decades for this specific form of cancer to develop. The impact of UV1 vaccination in patients with malignant pleural mesothelioma has been evaluated in a randomized Phase II clinical trial, NIPU. In the study, UV1 was combined with checkpoint inhibitors ipilimumab and nivolumab and compared to ipilimumab and nivolumab alone as a second-line treatment, after first-line treatment with platinum-based chemotherapy. The results from the study, presented at the European Society for Molecular Oncology (“ESMO”) Congress 2023, demonstrated a clinically meaningful improvement in overall survival for UV1 treatment in combination with ipilimumab and nivolumab versus ipilimumab and nivolumab alone. In addition, UV1 maintained its positive safety and tolerability profile in this combination with no added toxicities. Oslo University Hospital sponsored the NIPU study with support from Bristol Myers Squibb and Ultimovacs.
On February 5, 2024, the FDA granted Fast Track designation for UV1 in combination with ipilimumab and nivolumab for the treatment of patients with unresectable malignant pleural mesothelioma to improve overall survival. In October 2023, UV1 received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with mesothelioma. In December 2021, the FDA granted Orphan Drug Designation for UV1 for the treatment of stage IIB-stage IV melanoma. In October 2021, the FDA granted Fast Track designation for UV1 as add-on therapy to ipilimumab or pembrolizumab for the treatment of unresectable or metastatic melanoma.
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About NIPUNIPU (Nivolumab and Ipilimumab Plus/minus UV1 vaccination) is a randomized, multi-center phase II trial in which Ultimovacs’ universal cancer vaccine, UV1, is evaluated in combination with Bristol-Myers Squibb’s checkpoint inhibitors, nivolumab and ipilimumab, as second-line treatment of malignant pleural mesothelioma. The trial sponsor is Oslo University Hospital, supported in the preparation and execution of the trial by Ultimovacs and Bristol Myers Squibb. The 118 patients are randomized 1:1 into two treatment arms. All participants receive treatment with nivolumab (240 mg every two weeks) and ipilimumab (1 mg/kg every six weeks) until disease progression, unacceptable toxicity, or for a maximum of 2 years. Patients randomized to the experimental arm received eight intradermal injections of UV1 vaccine during the first three months of treatment. The objective of the study is to achieve a clinically meaningful progression-free survival (PFS) benefit in patients with malignant pleural mesothelioma (MPM) after progression on first-line standard platinum doublet chemotherapy. Subsequent events emerging in patients in both arms of the NIPU study will continue to be monitored beyond the read-out of the primary endpoint. The ipilimumab and nivolumab combination has been approved as first-line treatment for patients with malignant pleural mesothelioma in Europe and the U.S.
About Mesothelioma Mesothelioma is a rare and aggressive type of cancer that occurs in the thin layer of tissue that surrounds the lungs and inside of the chest. Mesothelioma accounted for 30 870 new cancer cases and 26 278 cancer deaths worldwide in 2020, according to International Agency for Research on Cancer (Globocan 2020). Pleural mesothelioma is a disease with a high unmet medical need, especially in industrialized countries. The median overall survival is approximately one year. Occupational asbestos exposure is the No. 1 cause of the disease, and several occupations, like firefighters, military veterans, construction, and industry workers, are at risk. This cancer usually takes several decades to develop after a person’s first exposure to asbestos. Most patients are diagnosed after age 70 because of the long latency period. Even though the use of asbestos to a large extent is banned in many countries today, new incidences of mesothelioma will continue to be a medical and public health challenge because of the long latency period typical of the illness. For patients with inoperable disease, few treatment options are available after first-line chemotherapy. The combination of ipilimumab and nivolumab has recently shown increased survival compared to standard chemotherapy, but most patients do not respond, and improvements are called for. Telomerase is expressed in mesothelioma cells and is therefore a relevant target for therapeutic vaccination.
About UltimovacsUltimovacs is a clinical-stage biotechnology leader in novel immunotherapeutic cancer vaccines with broad applicability. Ultimovacs’ lead candidate, UV1, is an off-the-shelf therapeutic cancer vaccine directed against human telomerase (hTERT), an antigen that is present in 85-90% of cancers in all stages of tumor growth. A broad clinical program, with Phase II trials in five cancer indications enrolling more than 670 patients, aims to demonstrate UV1’s impact in combination with other immunotherapies in multiple cancer types expressing telomerase and where patients have unmet medical needs. UV1 is a patented technology owned by Ultimovacs. In addition, Ultimovacs holds all rights of the proprietary TET technology platform for any possible future use of formulations in various solid tumor indications. The Company is listed on Euronext Oslo Stock Exchange (OSE:ULTI). About UV1 Phase II programThe immunotherapeutic off-the-shelf cancer vaccine UV1 is investigated in combination with checkpoint inhibitors in patients with various cancer indications with diverse tumor biology. The diversity of the UV1 Phase II program places Ultimovacs in a favorable position to capture the cancer vaccine’s potential broad applicability when combined with checkpoint inhibitors:
INITIUM: Evaluating UV1 in combination with ipilimumab and nivolumab as first-line treatment for patients with malignant melanoma. Enrollment of 156 patients completed in July 2022. Expected readout in March 2024. Sponsored by Ultimovacs.NIPU: Evaluating UV1 in combination with ipilimumab and nivolumab as second-line treatment for patients with malignant pleural mesothelioma. Enrollment of 118 patients completed in January 2023. Data presented at the ESMO Congress in October 2023, showed that UV1 as add-on to ipilimumab and nivolumab, demonstrated a statistically significant and clinically meaningful improvement of overall survival versus ipilimumab and nivolumab alone. 31% of the patients receiving UV1 vaccine experienced an objective response, compared to 16% in the control arm. The investigator-initiated trial is led by Oslo University Hospital and supported by Bristol-Myers Squibb and Ultimovacs.FOCUS: Evaluating UV1 in combination with pembrolizumab as first-line treatment for patients with head and neck cancer. Enrollment of 75 patients completed in August 2023, expected readout H2 2024. The investigator-initiated trial is led by Halle University in Germany, supported by Ultimovacs.DOVACC: Evaluating UV1 in combination with olaparib and durvalumab as maintenance therapy in non-BRCA mutated patients with advanced ovarian cancer. More than 40% of the 184 patients enrolled as of Q4 2023 reporting, expected readout H1 2025. The investigator-initiated trial is led by NSGO-CTU and supported by ENGOT, AstraZeneca, and Ultimovacs.LUNGVAC: Evaluating UV1 combined with cemiplimab as first-line treatment of non-small cell lung cancer patients. More than 15% of 138 patients enrolled as of Q4 2023 reporting, expected readout H1 2026. The investigator-initiated trial is led by Vestre Viken (Drammen Hospital) and supported by Ultimovacs.
About UV1UV1 is an off-the-shelf therapeutic cancer vaccine designed to induce a specific T cell response against telomerase. UV1 consists of long, synthetic peptides representing a sequence in the reverse transcriptase subunit of telomerase (hTERT), shown to induce CD4+ T cells. These CD4+ T cells have the potential to provide inflammatory signals and T cell support believed to be critical for triggering a strong anti-tumor immune response. Following intradermal injection, antigen presenting cells (APCs) in the skin are exposed to the vaccine peptides. These APCs will process the peptides, and present vaccine epitopes on Human Leukocyte Antigen (HLA) molecules to naïve T cells in the lymph nodes. Activated vaccine specific T cells will then enter the circulation and search for cells displaying their cognate antigen in the context of HLA molecules.
The UV1 peptides contain several epitopes, shown to be non-restrictive in terms of (HLA) alleles for presentation. It is therefore not required to perform HLA pre-screening of patients, which potentially enables broad population utilization of the vaccine. UV1 is administered over three months as eight intradermal injections together with the immune-modulator GM-CSF.
For further information, please see www.ultimovacs.com or contact:
Carlos de Sousa, CEO Email: carlos.desousa@ultimovacs.comPhone: +47 908 92507
Anne Worsøe, Head of IREmail: anne.worsoe@ultimovacs.comPhone: +47 90686815
This information is considered to be inside information pursuant to the EU Market Abuse Regulation and is subject to the disclosure requirements pursuant to Section 5-12 in the Norwegian Securities Trading Act.This stock exchange announcement was published by Anne Worsøe, Head of Investor Relations at Ultimovacs ASA, on February 19, 2024 at 07:00 CET.
