IO102: IO Biotech’s lead anti-cancer therapy targeting IDO

IO102 is a anti-cancer therapy containing a 21-mer IDO-derived peptide. it is a disruptive immune therapy with dual mode of action killing both cancer and normal cells with immune suppressive functions.

IO102 is a first-in-class IDO disruptive immune modulating anti-cancer therapy which potentially can be the ideal safe and effective add-on treatment to checkpoint inhibitors for multiple cancer indications. The basis for the discovery of IO102 was IDO101, a anti-cancer therapy containing a 9-mer IDO-derived peptide.

Clinical status

IO Biotech has entered into a clinical collaboration with MSD evaluating IO102 in combination with KEYTRUDA® (pembrolizumab) in first-line treatment of patients with metastatic non-small cell lung cancer. IO Biotech will maintain global commercial rights to IO102. The study is planned to be initiated in Q2, 2018.

Two Investigator Initiated Trials have been completed with IO Biotech’s IDO anti-cancer therapies

• Phase I NSCLC study completed (IO101), Q1 2014 – Superior OS in anti-cancer therapies treated cohort and long term survival (5 year follow up data).
• Phase I Melanoma study in combination with Ipilimumab completed, Q2 2016. Confirmed Safety.

The first-in-man clinical phase I vaccination trial using anti-cancer therapy encompassing an HLA-A2-restricted, 9 amino acid IDO-derived T-cell epitope was conducted. The study comprised 15 HLA-A2+ patients with advanced NSCLC who were vaccinated with IO101 in Montanide adjuvant following pre-treatment with topical Imiquimod (Aldara® Cream). The anti-cancer therapy was well tolerated with manageable side effects and no CTCAE grade 3/4 toxicities. One patient obtained an objective partial response after demonstrating continuous regression of liver metastases on anti-cancer therapy treatment for 1 year.

The study has been performed in 10 melanoma patients where treatment with IO102 was combined with treatment with the checkpoint inhibitor, ipilimumab (anti-CTLA4 antibody). There were no safety concerns as no treatment-related grade 3 or 4 toxicity was reported. Four patients had disease stabilization. At first evaluation, five of the ten treated patients were in stable disease, one of which had an unconfirmed partial response.

Preclinical and clinical data

In vitro and in vivo models demonstrate synergy of IO102 in combination with other immune modulatory drug e.g. anti-PD1 Ab, as well as other anti-cancer therapies. Clinical data from phase I NSCLC study (IO101) indicated superior overall survival in anti-cancer therapy treated cohort and long term survival (5 year follow up data). Safety was confirmed in Phase I Melanoma study in combination with Ipilimumab.

Publications by founders

Cytotherapy, August 2016: Safety, immune and clinical responses in metastatic melanoma patients vaccinated with a long peptide derived from indoleamine 2,3-dioxygenase in combination with ipilimumab