Cancer drug for children and young people with glioblastoma given to patients in study

September 2, 2022 - 3 minutes
Picture/Institut Curie

Clinical-stage bio company, Onxeo, has announced that the first patients have been enrolled and treated in a phase 1b/2 clinical study.

The study is to evaluate the efficacy and tolerability of AsiDNA2, a DDR inhibitor combined with radiotherapy in children, adolescents or young adults with relapsed high grade glioma (HGG).

Onxeo, which focuses on drugs targeting tumor DNA damage response (DDR) in particular against rare or resistant forms of cancer says the therapy offers the hope of providing better care and treatment for these high-risk pediatric cancers.

Poor prognosis

HGG, which represents approximately 20% of tumors of the central nervous system (CNS) in children, continue to have a very poor prognosis with a five-year-survival rate of less than 20%.

Surgery combined with radiotherapy or chemotherapy often allows the disease to be controlled. However, this disease control is not durable due to the development of treatment-resistant tumor cells.

Studies conducted in preclinical models as well as initial clinical trials conducted in adults have highlighted AsiDNA’s synergetic effect when combined with treatments that target and destroy the DNA, such as radiotherapy.

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Institut Curie, Paris

This phase 1b/2 trial, sponsored by Paris-based Institut Curie, is being conducted within the framework of the European ITCC4 consortium, in order to evaluate AsiDNA’s efficacy and tolerability by enrolling a maximum of 32 patients made up of children, adolescents or young adults with relapsed HGG.

A first patient has been enrolled in the study at the Institut Curie’s SIREDO Center. Additionally, other French centers as well as European countries are planned to be initiated in the coming months.

The study’s first interim results are projected in the first quarter of 2024.

Shefali Agarwal, Onxeo’s president and CEO, said: “Onxeo is proud of its clinical collaboration with Institut Curie, and would like to thank Professor François Doz – the study’s lead investigator – for his involvement in this project. We hope that the combination of AsiDNA and radiotherapy in this type of cancer will provide a real benefit to patients suffering from a recurrent form of high-grade glioma.”

European Fight Kids Cancer

Doz is a pediatric oncologist, deputy head of clinical research, innovation and teaching at Institut Curie’s SIREDO Center and this study’s lead investigator. This phase 1b/2 study is supported by a grant from the European Fight Kids Cancer program.

He said: “The enrollment of the first patient is a major step in this proof-of-concept study whose goal is to evaluate the efficacy and good tolerability of the systemic administration of AsiDNA in combination with a new radiotherapy. We hope that this study will provide encouraging evidence enabling an improvement in the prognostic outcome of a disease that is a key unmet need.”

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