The Food and Drug Administration (FDA) grants IO Biotech breakthrough therapy designation for IO102 and IO103 in combination with anti-PD-1 in unresectable/metastatic melanoma

Dec 15 2020

Copenhagen, Denmark – December 15, 2020:  IO Biotech today announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation for a combination of the potential therapy IO102 and IO103 with anti-PD-1 mAb for patients with unresectable/ metastatic melanoma.

IO102 and IO103 are IO Biotech’s lead immuno-oncology candidates. Both compounds are based on IO Biotech’s proprietary T-win® technology platform which enables the identification of compounds with a dual mechanism of action targeting and directly killing immunosuppressive cells and tumor cells while indirectly activating other T-effectors, leading to strong anti-tumor responses without adding additional safety concern. Specifically, IO102 and IO103 are first-in-class, immune modulatory vaccines designed to engage and activate IDO and PD-L1 specific human T-cells.

The FDA decision to grant breakthrough therapy was based on data from the MM1636 Phase 1/2 clinical trial with 30 patients with metastatic melanoma receiving IO102, IO103 and anti-PD-1. According to the data recently presented in a late-breaking abstract at the European Society for Medical Oncology (ESMO) Virtual Congress 2020, the combination of IO102 and IO103 vaccines and nivolumab was shown to be safe with encouraging early efficacy data; an overall response rate (ORR) of 79 percent was reached and 45 percent of patients achieved a complete response (CR), or complete disappearance of their tumors. Vaccine specific T-cells were located in the peripheral blood mononuclear cells (PBMCs) and at the tumor site.  

“A breakthrough designation is an expedited program in which FDA will work closely with IO Biotech to provide guidance on subsequent development of “Synthetic Peptide Vaccines (IO103) Encoding Human PD-L1 (9-27) and (IO102), targeted to IDO, Indoleamine 2,3-dioxygenase and anti-PD-1″ for treatment of unresectable/metastatic melanoma, including providing advice on generating evidence needed to support approval of the drug in an efficient and potentially expedited manner”,” said Mai-Britt Zocca, PhD, Chief Executive Officer and founder of IO Biotech. “This is an important achievement for IO Biotech’s clinical program and we are committed to bring this forward to patients as soon as possible.”

About MM1636 trial
The MM1636 trial is an investigator-initiated trial at the Copenhagen University Hospital, Herlev which entered 30 patients with metastatic melanoma. In this Phase 2 clinical trial, patients were treated with IO Biotech’s multi antigen vaccine, IO102-IO103 in combination with anti-PD-1 antibody, as first line treatment. IO102-IO103 are administered every second week until 12 weeks and thereafter every fourth week up to one year. The trial objectives are to assess safety, immune responses in blood and biopsies as well as efficacy.

About Melanoma
Globally, about 290,000 new cases of cutaneous melanoma, the most aggressive type of skin cancer, are diagnosed and annually more than 60,000 will die (Bray et al. 2018). The incidence of cutaneous melanoma is increasing (Forman et al. 2014) and advanced melanoma (unresectable or metastatic) will have a fatal outcome if left untreated. The median overall survival (OS) in patients with stage IV melanoma (untreated or treated with BRAF inhibitor and MEK inhibitor) is between 22-25 months and a 3-5-year OS is around 40% (Ascierto et al. 2016; Long et al. 2017).

Therefore, there is a clear unmet need for improved combination therapies enhancing anti-PD-1 efficacy without a significant increase in toxicity.

About IO Biotech
IO Biotech is a clinical stage biotech company developing disruptive immune therapies for the treatment of cancer. The pipeline of first-in-class immune modulating anti-cancer therapies is developed by a unique technology platform, T-win®, enabling the activation of T cells that are specific for immune-suppressive molecules. IO Biotech has a proven track record of progressing preclinical and clinical compounds. The two lead compounds targeting IDO and PD-L1 are in clinical development and several pipeline compounds are in pre-clinical phase. For further information, please visit

Mai-Britt Zocca, PhD, CEO and founder of IO Biotech       E-mail: