Cytovation to present full safety and efficacy data from the CICILIA Phase I/IIa trial evaluating CY-101 in solid tumors at ESMO 2024

• CY-101 well tolerated with early signs of clinical activity, especially in tumors with dysregulated Wnt/β-catenin signalling
• CY-101 granted Orphan Drug Designation in US for the treatment of Adrenocortical Carcinoma, a Wnt/β-catenin driven tumor type

Bergen, Norway, September 13, 2024 – Cytovation ASA, a clinical stage oncology company focused on the development of its first-in-class bifunctional immunotherapy CY-101 (CyPep-1) announces that it will deliver a poster presentation at ESMO 2024 entitled “Safety and activity of CY-101 in patients with advanced solid tumors: the Phase I/IIa CICILIA trial.” Data from this study, evaluating intratumoral (IT) administration of CY-101 monotherapy and in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with heavily pretreated advanced solid tumors, show that CY-101 is well tolerated at the recommended Phase 2 dose (RP2D) of 20 mg. Furthermore, these data demonstrate early signs of antitumor activity, especially in tumors with dysregulated Wnt/β-catenin signaling.

CY-101 is a synthetic peptide with a dual mode of action that both inhibits the oncogenic Wnt/β-catenin pathway by activating Axin2 and has a pore-forming membranolytic effect on cancer cells that exposes antigens and triggers a systemic tumor-specific immune response. Interim results from CICILIA were reported in 2023 (link to press release here) confirming that all trial endpoints were met, with CY-101 demonstrating a consistent safety profile across tumor types and strong early signals of efficacy.

Data from the ESMO poster show that no dose-limiting toxicities were observed in the dose- escalation part of the study with the RP2D defined at 20 mg. Analyses of paired tumor biopsies demonstrated induction of cancer cell death in >70% across tumor types at the RP2D, translating to a clinical benefit in several different cancer types. Notably, among six patients with adrenocortical carcinoma (ACC) receiving CY-101 monotherapy, a disease control rate of 50% (n = 3/6) was observed, with durable responses (> 6 months) in two patients. Both patients expressed β-catenin and had somatic mutations in the Wnt/β-catenin pathway.

Separately, the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to CY-101 monotherapy for the treatment of ACC. Orphan Drug Designation is granted to investigational therapies that are intended for the treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the US. Orphan Drug Designation provides several benefits to drug developers, including certain development cost benefits in the US, increased FDA interaction and eligibility for seven-year market exclusivity following approval.

“We believe CY-101 is a potentially important new treatment option that offers a truly differentiated approach to targeting Wnt/β-catenin driven cancers, an oncogenic pathway dysregulated in more than 20% of cancers,” said Lars Prestergarden, CEO of Cytovation. “Our early data clearly confirm that CY-101 warrants further investigation in larger studies, and we are very pleased to receive Orphan Drug Designation for CY-101 in ACC. We look forward to advancing towards our planned Phase 2 trial in ACC with registrational intent.”

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merk & Co., Inc., Rahway, NJ, USA.

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About Adrenocortical Carcinoma (ACC)

ACC, also known as adrenal cancer, is a rare cancer with a very poor prognosis and few treatment options. In the metastatic setting there is no approved treatment option after first line.

About Cytovation 

Cytovation ASA is a clinical stage immune-oncology company focused on the development of CY-101, a first-in-class bifunctional immunotherapy.

CY-101 has a unique dual mechanism of action, specifically eliminating cancer cells by targeting the cell membrane and releasing neo-antigens, and by inhibiting the Wnt/β-catenin oncogenic pathway to restrict tumor growth and reverse immune exclusion associated with β-catenin expression. Dysregulation of this pathway has been associated with several different cancer types including colon, liver, uterine, lung and ovarian cancer, among others. This dual mode of action induces a systemic, tumor-specific immune response.

For more information, please visit www.cytovation.com.