invIOs reports promising Phase 1b results for cell therapy APN401 in advanced solid tumors

invIOs reports promising Phase 1b results for cell therapy APN401 in advanced solid tumors

• Successful completion of the multi-center trial with autologous cell therapy APN401 marks a key step forward toward clinical use.
• APN401 shows a favorable safety profile and promising efficacy signals in patients with late-stage solid tumors.
• The observed immune biomarker signature following APN401 treatment allows risk-benefit assessment for further clinical development.

Vienna, Austria, 30 June 2025: invIOs GmbH (“invIOs”), a privately held biotechnology company focused on developing innovative cancer immunotherapies, today announced the completion of the Phase 1b trial of APN401, a novel autologous cell therapy based on Cbl-b silenced peripheral blood mononuclear cells (PBMCs).

APN401 leverages invIOs’s proprietary EPiC cell therapy platform to modify patients’ own PBMCs by blocking Cbl-b expression. Cbl-b belongs to so-called immune checkpoint molecules, primarily upregulated in immune cells that have exhausted their capacity to eliminate tumor cells.

This study was performed in Austria utilizing two GMP-compliant manufacturing sites for assessing two dosing levels of APN401 across four clinical study centers. It evaluated APN401’s safety, tolerability, and initial signs of efficacy in patients with advanced solid tumors who have exhausted standard treatment options.

A total of 11 patients with advanced solid tumors, including head and neck cancer, colorectal cancer, uveal melanoma, and small cell lung cancer, were enrolled. All patients were heavily pretreated with at least two prior lines of therapy. Disease stabilization was achieved in 37.5% of patients, which was accompanied by strong expansion of individual T cells and increased plasma levels of biomarkers associated with cytotoxic T cell activity. Although tumor responses were modest, the achievement of a 7.8-month median overall survival in a population with no remaining treatment options is regarded as a noteworthy clinical outcome.

Romana Gugenberger, PhD, Chief Medical and Scientific Officer of invIOs, commented: “The successful completion of the Phase 1b trial with APN401 validates the potential of our EPiC platform to empower autologous cells for targeted cancer cell killing. Building on these encouraging results, we are advancing the development of INV441, which uses tumor-infiltrating lymphocytes and leverages the same underlying technology as APN401. INV441 is on track to enter its first-in-human clinical trial for the treatment of glioblastoma, an aggressive form of brain cancer that lacks effective treatments. Together with our other programs, including the novel immune-activating small molecule INV501, we remain confident in our ability to deliver new treatment options for patients with hard-to-treat tumors.”

About invIOs

invIOs is a privately held biotech company based in Vienna, Austria, focusing on the discovery and development of novel therapies for cancer. The invIOs team applies its extensive knowledge of immune system activation to create personalized and targeted therapies for solid tumors. The company is progressing a pipeline of four programs that include both small molecule and cell therapy strategies. invIOs is also partnering with leading institutions and universities—such as the Dana-Farber Cancer Institute—to co-develop treatments for glioblastoma. EPiC, invIOs’s proprietary cell therapy platform, enables rapid treatment of patients in an out-patient setting. EPiC is a technology platform that allows the creation of personalized cell therapies based on localized processing of a patient’s fresh immune cells.

The platform’s lead asset, APN401, has successfully completed Phase 1 clinical trials in patients with various solid tumors. The second candidate, INV441, is currently being developed for glioblastoma and is on track to enter a first in human Phase 1 clinical trial. Additionally, INV451, the third EPiC-based program, represents a next-generation CAR-T cell approach targeting rare tumor types.

INV501 is a novel small molecule candidate that can selectively enhance anti-tumor immune responses. It induces T cell activation, proliferation, and tumor-specific cytotoxicity. An orally available compound with favorable bioavailability, INV501 is currently in preclinical testing in several solid tumor models and has demonstrated preclinical proof of concept with strong data in multiple solid tumor indications.

For further information, please visit www.invios.com and connect with us on LinkedIn.

Contacts

invIOs
Peter Llewellyn-Davies
CEO/CFO
T +43 1 8656577 0
Email: investors@invios.com

MC Services AG
Dr. Cora Kaiser, Dr. Regina Lutz
T +49 89 210 228 0
US: Laurie Doyle
T +1-339-832-0752
Email: invios@mc-services.eu