Ultimovacs receives Orphan Drug Designation from EMA
22 February, 2024
Ultimovacs has been granted Orphan Drug Designation from the European Medicines Agency for its universal cancer vaccine, UV1, as a treatment for mesothelioma, a rare and aggressive form of cancer with limited therapeutic options. The designation was granted based on results from the phase II clinical trial NIPU and brings regulatory and commercial benefits to the development of the immunotherapy. BioStock reached out to CEO Carlos de Sousa for a comment.

Ultimovacs is a biotech company developing novel immunotherapeutic cancer vaccines. Its lead candidate, UV1, is a universal cancer vaccine aimed at boosting the patient’s immune system to slow down cancer progression. UV1 is currently being evaluated in various cancer indications: malignant melanoma, malignant pleural mesothelioma, ovarian cancer, head and neck cancer and non-small cell lung cancer.

Orphan Drug Designation in both the US and EU
Both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have established Orphan Drug Designation (ODD), a programme to facilitate the development of orphan drugs – medicines that are being developed for the treatment of rare and life-threatening conditions. In the EU, a disease is defined as rare if it affects fewer than 5 in 10,000 people across the EU.

ODD provides companies with certain benefits and incentives, including ten years of market exclusivity upon approval, clinical protocol assistance, access to a centralised marketing authorisation procedure, and reduced regulatory fees.

Ultimovacs’ candidate UV1 has previously been granted ODD from the FDA in treating patients with mesothelioma and malignant stage IIB-stage IV melanoma. On February 19, the company announced that EMA had issued a positive opinion on the ODD application for UV1 in mesothelioma. CEO Carlos de Sousa commented on the positive notice in a press release:

»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. 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.«

NIPU showed improved overall survival in mesothelioma
The ODD will facilitate the continued development of UV1 in mesothelioma, a rare and aggressive type of cancer that occurs in the thin layer of tissue that surrounds the lungs and inside of the chest. Patients with this cancer form have a high mortality rate and few therapeutic options.

The designation was granted based on the results from the randomised phase II clinical trial, NIPU, evaluating UV1 as an add-on to Bristol Myers Squibb’s checkpoint inhibitors nivolumab and ipilimumab in patients with malignant pleural mesothelioma. The NIPU study showed that the combination treatment gives a statistically significant and clinically meaningful improvement of overall survival compared to ipilimumab and nivolumab alone.

Ultimovacs also recently received FDA Fast Track designation for UV1 in patients with unresectable malignant pleural mesothelioma. This will further facilitate the development in this indication by more frequent interactions with the FDA.

CEO comments
BioStock reached out to CEO Carlos de Sousa to learn more about the importance of the ODD for UV1 in mesothelioma.

Carlos de Sousa, vd Ultimovacs
Carlos de Sousa, CEO Ultimovacs
Congratulations on receiving ODD for UV1 in mesothelioma from the EMA! Can you share your initial reaction to this designation and its significance for Ultimovacs?
– We are very excited about this news as this is the first designation we have received from the EMA. It is a great validation of the potential UV1 holds for this hard-to-treat cancer and shows that the EMA recognises the importance of finding new therapeutic options for mesothelioma. The designation is an important tool to support the drug development for rare disease indications with a high unmet medical need. It enables us to stay in a close dialogue with the EMA on the next steps for the programme.

ODD offers several regulatory and commercial benefits, such as market exclusivity and reduced regulatory fees. How does Ultimovacs plan to leverage the ODD to advance the development of UV1 in mesothelioma?
– With the ODD, we have the opportunity to more frequently interact with the regulatory agency and therefore closely align on a potential clinical trial design and the requirements for a future market approval. We are currently waiting for the lead investigators from the Oslo University-initiated programme to present more detailed and mature data, so we can assess how we are best moving this programme forward.

Looking ahead, what milestones can we expect from Ultimovacs regarding the development in mesothelioma and other cancer indications?
– This March, we are expecting an important formative milestone for Ultimovacs with the topline data readout from our own INITIUM trial. This randomised phase II study evaluates UV1 vaccination in combination with ipilimumab and nivolumab for unresectable or metastatic malignant melanoma. We are very much looking forward to these results and presenting them to the scientific community and our shareholders. The data could be the first to demonstrate benefit of an off-the-shelf cancer vaccine over standard of care in a randomised trial in advanced melanoma, which represent a major step forward for Ultimovacs and the field of cancer treatment.

The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.
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