Ultimovacs Receives FDA Orphan Drug Designation for the UV1 Cancer Vaccine for Treatment of Mesothelioma

• The orphan drug designation was granted based on data from the randomized Phase II clinical trial NIPU
• The results from the NIPU study will be presented at the ESMO Congress being held October 20-24, 2023 in Madrid
• UV1 also received FDA orphan drug designation for treatment of patients with malignant melanoma in December 2021

Oslo, October 09, 2023: Ultimovacs ASA (“Ultimovacs”) (OSE ULTI), a clinical-stage biotechnology leader in novel immunotherapeutic cancer vaccines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the company’s therapeutic cancer vaccine UV1 for the treatment of patients with mesothelioma. The designation was granted based on the initial data from the Phase II clinical trial, NIPU.

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 occupationally or environmentally 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 is being assessed in the 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 randomized, open-label, multicenter trial with 118 patients was conducted in Australia, Denmark, Norway, Spain, and Sweden. The first patient in the trial was enrolled in June 2020, and the last patient was enrolled in January 2023. The NIPU study is sponsored by Oslo University Hospital with support from Bristol-Myers Squibb and Ultimovacs.

The results from the study will be shared at the ESMO Congress in Madrid, held October 20-24, in an oral presentation by the Principal Investigator, Åslaug Helland, MD, Ph.D., Professor at Oslo University Hospital. The presentation 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”.

“Gaining FDA orphan drug designation for UV1 in mesothelioma highlights UV1’s potential and the significant need for new treatment options for this patient population,” said Carlos de Sousa, CEO of Ultimovacs. “We look forward to the presentation of the NIPU results at the ESMO Congress later this month and to continue our dialogue with the FDA as we seek to bring UV1 to cancer patients as quickly as possible.”

The FDA’s Office of Orphan Products Developments grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including potentially up to seven years of market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.

UV1 is a therapeutic cancer vaccine that generates an immune response against the human telomerase (hTERT) enzyme.  The enzyme is essential for the ability of cancer cells to proliferate. Telomerase is present in 85-90% of all cancers across all stages of the disease.

Ultimovacs is evaluating the universal cancer vaccine UV1 in a broad clinical development program across various cancer indications with different biologies and disease stages, combined with different checkpoint inhibitors. The topline data from NIPU are the first results among the currently five randomized trials in the UV1 Phase II clinical program. In addition to malignant pleural mesothelioma, Phase II studies are ongoing in patients with malignant melanoma, head and neck cancer, ovarian cancer, and non-small cell lung cancer. The topline data from the malignant melanoma and head and neck cancer trials are also expected within a year. UV1 is a patented, proprietary technology owned by Ultimovacs.

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About NIPU
NIPU (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 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 surrounding the lungs and inside the chest. Mesothelioma accounted for 30 870 new cancer cases and 26 278 cancer deaths worldwide in 2020, according to the 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. Few treatment options are available after first-line chemotherapy for patients with inoperable disease. 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 the UV1 Phase II program
The immunotherapeutic 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. Expected readout H1 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; results will be presented at the ESMO Congress in October 2023. The investigator-initiated study 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, expected readout H2 2024. The investigator-initiated study 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. >20% of 184 patients recruited as of Q2 2023 reporting, expected readout H2 2024. The investigator-initiated study 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. <10% of 138 patients recruited as of Q2 2023 reporting, expected readout H2 2025. The investigator-initiated study is led by Vestre Viken (Drammen Hospital) and supported by Ultimovacs.

About Ultimovacs
Ultimovacs is a clinical-stage biotechnology leader in novel immunotherapeutic cancer vaccines with broad applicability. Ultimovacs’ lead cancer vaccine candidate UV1 is directed against human telomerase (hTERT), an antigen 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 universal, off-the-shelf and easy to use, and is a patented technology owned by Ultimovacs.
In addition, Ultimovacs’ adjuvant platform, based on the proprietary Tetanus-Epitope-Targeting (TET) technology, combines tumor-specific antigens and adjuvant in the same molecule and is in Phase I clinical development.

About UV1
UV1 is a universal 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 is 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.com
Phone: +47 908 92507

Anne Worsøe, Head of IR
Email: anne.worsoe@ultimovacs.com
Phone: +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 October 9, 2023 at 07:00 CET.