Development stage: Phase II

A recombinant fusion protein that links the monoclonal anti-GD2 antibody hu14.18 with interleukin-2 (IL2) for the treatment of relapsed/refractory high-risk neuroblastoma and other cancers.

APN301 is a fusion protein of a humanized antibody (hu14.18) and IL2. The antibody portion specifically binds to the GD2 antigen that is strongly expressed on neuroblastoma and several other cancers. IL2 is a cytokine that recruits multiple immune effector cell types. In patients, APN301 is designed to localize GD2-positive tumor cells via the antibody component. The fused IL2 then stimulates the patient's immune system against the tumor by activation of both, NK and T cells, whereas the Fc portion of the antibody is designed to trigger tumor cell killing by antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity. The immunocytokine has shown certain activity in a Phase II clinical study in children with relapsed/refractory neuroblastoma. It was also tested in a Phase I/II study in late stage malignant melanoma, showing immune activation. Due to the recent success with APN311 (marketing authorization in the EU for the treatment of neuroblastoma) the development focus for APN301 has changed. The biologic will now be investigated in melanoma and GD2+ pediatric cancers other than neuroblastoma. In this regard a new application (intratumoral injection) will be investigated leading to a "in situ vacctination" effect.



Shusterman et al. Antitumor Activity of Hu14.18-IL2 in Patients With Relapsed/Refractory Neuroblastoma: A Children’s Oncology Group (COG) Phase II Study. J Clin Oncol. 2010 Nov 20;28(33):4969-75

Morris et al. In Situ Tumor Vaccination by Combining Local Radiation and Tumor-Specific Antibody or Immunocytokine Treatments. Cancer Res. 2016 Jul 1;76(13):3929-41