invIOs publishes positive clinical data on CBL-B-silenced autologous cell therapy APN401 in peer-reviewed journal

invIOs GmbH (“invIOs”), a privately held biotechnology company developing innovative cancer immunotherapies, today announced the publication of new clinical data with its autologous PBMC (peripheral blood mononuclear cell) therapy APN401 in the peer-reviewed journal, Molecular Therapy Oncology. The publication, “Phase I Evaluation of APN401, an Autologous CBL-B–Silenced PBMC Therapy, in Advanced Solid Tumors”, reports findings from two Phase 1 studies evaluating APN401 in patients with advanced, treatment-refractory solid tumors.

A total of 17 patients received APN401 across three dose levels. The patients had advanced solid tumors and had received between two and eleven prior lines of systemic therapy. The treatment was well tolerated, with no dose-limiting toxicities, cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, or grade ≥ 3 treatment-related adverse events. The highest dose of 4.5 × 10⁷ PBMCs/kg was selected as the recommended Phase 2 dose.

Disease stabilization was observed in 5 out of 10 RECIST-evaluable patients. Exploratory immune profiling identified systemic changes following APN401 treatment, including increased levels of cytotoxic mediators and expansion of specific T-cell populations. These changes were most pronounced in patients with stable disease. This suggests that APN401 can enhance immune activity against the tumor and that this effect may be linked to clinical benefit.

APN401 is an autologous cell therapy utilizing patients’ own PBMCs in which the intracellular immune checkpoint CBL-B is transiently silenced using siRNA. CBL-B acts as a brake on the immune system: it negatively regulates antigen receptor signaling and T-cell activation. By temporarily reducing CBL-B activity in immune cells, APN401 cell therapy is engineered to overcome the immunosuppressive tumor microenvironment and attack tumor cells.

We are thrilled to see our clinical data with APN401 published in a peer-reviewed journal. We are encouraged by the favorable safety profile, the disease stabilization observed, and the systemic immune changes following treatment. This is especially noteworthy considering that these patients had already received multiple prior lines of therapy. These results highlight the promise of CBL-B silencing as a new approach in immuno-oncology and demonstrate that our EPiC cell manufacturing platform can reliably produce cell therapies through a rapid, decentralized manufacturing process. Together, these findings support further clinical evaluation of APN401 and our EPiC platform as we work towards new treatment options for patients with difficult-to-treat solid tumors.”

Romana Gugenberger, PhD, Managing Director, invIOs GmbH

APN401 is produced using invIOs’ proprietary EPiC^® point-of-care platform, which allows for manufacturing of the cell therapy product with a vein-to-vein time of less than 24 hours. The study demonstrated reproducible manufacturing across sites and supported the feasibility of repeated administration.