Project Overview

APEIRON’s product pipeline includes five immune-oncology (IO) product candidates in clinical and discovery stages, and one marketed product that was licensed to a partner: 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.

GD2 Target

APN311 is a therapeutic antibody to treat pediatric patients with neuroblastoma, exclusively outlicensed to and marketed by EUSA Pharma as Qarziba®.
APN301 is an immunocytokine consisting of a humanized antibody genetically linked to two human IL-2 molecules. The antibody part of APN301 specifically binds to disialoganglioside GD2, which is overexpressed in tumors of neuroectodermal origin, including sarcoma and melanoma. APN301 is designed to target GD2-positive tumor cells and stimulate the activation of immune effector cells such as natural killer (NK) and T cells, leading to tumor cell death via antibody-dependent cellular cytotoxicity (ADCC), complement-dependent cytotoxicity (CDC), and tumor-specific T cell response. Intravenous administration of APN301 has been evaluated in several Phase 1 and 2 clinical studies in advanced cancers.

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.

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.

Novel IO Targets

APEIRON`s novel immune oncology projects are directed against novel undisclosed targets and are being developed to treat tumors with substantial unmet medical need.
APN411 is a joint project from APEIRON’s IO alliance with Sanofi and Evotec and is an oral New Chemical Entity (NCE) portfolio of small molecule compounds against an undisclosed IO target.
APN421 is an oral NCE discovery program against an undisclosed novel target to enhance the activity of human immune cells such as CD4+ and CD8+ T cells as well as NK cells that may have therapeutic utility as a novel cancer immunotherapy.

Non-Oncology Programs

APN01 is a recombinant human Angiotensin Converting Enzyme 2 (rhACE2). This program was out-licensed to GlaxoSmithKline (GSK), which is currently conducting clinical studies in ALI (Acute Lung Injury) and PAH (Pulmonal arterial hypertension).