APEIRON’s product pipeline includes five immune-oncology (IO) product candidates in clinical and discovery stages, and one commercial stage product: the Cbl-b projects (APN401, APN431), the GD2 programs (APN311, APN301) and the novel IO discovery projects (APN411, APN421). Our non-IO product APN01, a recombinant enzyme of the Renin Angiotensin System, was out-licensed to GlaxoSmithKline which further validates APEIRON’s robust pipeline.
APN311 is a therapeutic antibody to treat pediatric patients with neuroblastoma, exclusively outlicensed to and marketed by EUSA Pharma as Qarziba®.
|Cbl-b Checkpoint Inhibition
APN401, a first-in-class autologous cellular therapy. Cbl-b represents a novel class of intracellular checkpoints as opposed to the immune checkpoint molecules PD-1/PD-L1 and CTLA-4. APN401 is designed to silence Cbl-b mRNA ex vivo in autologous peripheral blood mononuclear cells (PBMCs). These engineered autologous PBMCs are then reinfused into the patient with the entire procedure conducted in an ambulatory setting. APN401 is well tolerated and has a favorable safety profile and has shown preliminary evidence of clinical activity in patients with advanced solid tumors in current Phase 1 trials. Preparations for clinical studies in several solid tumor indications are ongoing.
APN431 is based on short cell-penetrating peptides which inhibit Cbl-b protein. Cbl-b plays a role in both the adaptive and the innate immune system and therefore can effectively enhance anti-tumor activities when knocked-out/silenced/inhibited. APN431 was in-licensed from the Institute of Molecular Biotechnology (IMBA) in Vienna and the Medical University in Vienna (MUW) in June 2018. The peptide-based inhibition by APN431 represents a novel approach to target the intracellular Cbl-b protein in immune cells of patients with various cancers.
APEIRON`s discovery projects are directed against novel undisclosed targets and are being developed to treat tumors with substantial unmet medical need.