Our scientific development comprises four platforms, namely the GD2 target, the respiratory platform around ACE2, the next-generation checkpoint inhibition Cbl-B and novel IO targets.

GD2 Target

APN311 is a therapeutic antibody to treat pediatric patients with neuroblastoma, exclusively outlicensed to and marketed by EUSA Pharma as Qarziba®.

Respiratory platform around ACE2

APN01 is a recombinant human Angiotensin Converting Enzyme 2 (rhACE2) under Phase-2 clinical development in ALI (Acute Lung Injury) and PAH (Pulmonal arterial hypertension). Recently, ACE2 has been shown to be the cellular entry receptor for the novel coronavirus SARS-CoV-2. APEIRON initiated a clinical Phase II study in several European countries with patients severly infected with SARS-CoV-2 in April 2020.

APN01 has a unique effect as it blocks infection and reduces inflammation

Next-Generation Checkpoint Inhibition

Cbl-b represents a novel class of intracellular checkpoints as opposed to the immune checkpoint molecules PD-1/PD-L1 and CTLA-4.Cbl-b represents a novel class of intracellular checkpoints as opposed to the immune checkpoint molecules PD-1/PD-L1 and CTLA-4.

The Cbl-b inhibition restores anti-tumor immune responses which lead to tumor destruction

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.

APN401is an ambulatory siRNA-based cell therapy

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.

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.

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.