Researchers at the Luxembourg Institute of Health (LIH) have played a pivotal role in advancing the clinical development of CY-101, a groundbreaking cancer therapy pioneered by Norwegian biotech company Cytovation AS

Thanks to the joint efforts of Cytovation and the LIH Tumor Immunotherapy and Microenvironment (TIME) Group, CY-101 has shown exceptional promise in both preclinical and clinical studies, resulting in its designation as an Orphan Drug by the U.S. Food and Drug Administration (FDA), a significant milestone in the advancement of new treatments for rare cancers. This achievement was made possible via a strong partnership with Cytovation, along with valuable support from the EU-Eurostars program
and the Luxembourg National Research Fund (FNR).

Adrenocortical cancer (ACC) is a rare and highly aggressive endocrine cancer with a poor prognosis, characterized by a 5-year survival rate of less than 60%. Furthermore, treatment options are limited, particularly for patients with metastatic ACC. Currently, the only approved therapy for ACC is Mitotane, which has notable limitations. Its efficacy is limited, with response rates of only 30%, and its significant systemic toxicities make it difficult for patients to tolerate.

CY-101, a novel immunotherapy, has demonstrated impressive efficacy against cancers such as ACC, melanoma, colorectal cancer, and hepatocellular carcinoma, all of which share common oncogenic processes. Notably, CY-101 has shown remarkable clinical results in ACC patients, offering new hope for those facing this challenging disease.
Within the framework of the EU Eurostars and FNR co-funded project, the joint efforts of Cytovation and the TIME Group led to the successful submission of an application to the FDA for Orphan Drug Designation (ODD) for CY-101. In July 2024, the FDA officially granted this designation, highlighting the potential of CY-101 to address a significant unmet medical need. The ODD status provides key advantages for CY-101, including access to expedited regulatory process, further accelerating its clinical development and bringing this promising therapy closer to patients in need.

“Securing FDA Orphan Drug Designation for CY-101 marks a significant milestone in our mission to develop innovative cancer treatments. Our collaboration with Cytovation has been crucial in advancing this therapy toward patients who lack effective treatment options. The promising results reinforce our confidence in the potential of CY-101 to make a difference in transforming treatments for ACC and other similar cancers,” said Dr. Bassam Janji, Group Leader of the TIME Group at the LIH.

“This achievement also underscores the ability of the LIH to conduct world-class translational research, positioning Luxembourg’s research landscape at the forefront of global cancer research. Through this milestone, the LIH demonstrates its commitment to innovative approaches, fostering the rapid translation of new discoveries into tangible clinical treatments and bridging the gap between cutting- edge research and real-world applications,” added Dr. Janji.

Based on this regulatory success, Cytovation has joined forces with Cancer Research UK and the Norwegian Cancer Society to further evaluate CY-101 in advanced clinical trials across a broader range of cancer types. This partnership is a critical turning point in advancing this transformative therapy, ensuring it reaches patients with the most urgent and unmet medical needs.