Child with cancer
Neuroblastoma is a rare type of cancer usually diagnosed in children under 2 years of age, and for which age-specific therapeutic options are needed.

The University of Birmingham and Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of orphan paediatric medicines, are pleased to announce the extension of their collaboration to leverage patient-level data of the BEACON refractory or relapsed neuroblastoma trial to support the marketing authorisation of Kizfizo.

Neuroblastoma is a rare type of cancer usually diagnosed in children under 2 years of age, and for which age-specific therapeutic options are needed.

The support of the University of Birmingham has been invaluable to us. The BEACON data has already demonstrated a clear clinical benefit and an improved overall survival over historical controls.

Jeremy Bastid, Chief Development Officer at Orphelia Pharma

Kizfizo (temozolomide oral suspension, 40 mg/ml) is the first ready-to-use oral liquid paediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carries a very poor prognosis.

The BEACON trial (NCT02308527) was a hypothesis-generating phase II trial that served to identify active regimens in relapsed or refractory neuroblastoma, some of which are now being further investigated. The trial recruited paediatric patients between 2013 and 2021 and involved the University of Birmingham as the sponsor, the European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

Kizfizo was not available in time for the BEACON trial but Orphelia Pharma and the University of Birmingham are collaborating to assess the clinical benefit and the safety of temozolomide-based regimens in relapsed or refractory high-risk neuroblastoma patients. Overall, anonymised data from the BEACON trial are being used to support the registration dossier of Kizfizo, according to a data sharing agreement drawn up by University of Birmingham Enterprise.

BEACON-2 is the follow-on trial that will test bevacizumab and dinutuximab beta in combination with the backbone chemotherapy temozolomide and irinotecan. As part of its ongoing commitment to child cancer, Orphelia Pharma has also agreed to support the new BEACON-2 trial, also sponsored by University of Birmingham, by making KIZFIZO available as a child-friendly temozolomide option, in addition to the standard form of temozolomide, where participating investigators can have access to this medicine upon regulatory approval of its use as part of the BEACON-2 trial.

Jeremy Bastid, Chief Development Officer at Orphelia Pharma, commented: “The support of the University of Birmingham has been invaluable to us. The BEACON data has already demonstrated a clear clinical benefit and an improved overall survival over historical controls.”

Dr Lucas Moreno, Head of Paediatric Haematology and Oncology at Vall d'Hebron University Hospital, Barcelona, Spain and Chief Investigator for BEACON said: “We are delighted that the data generated could not only delineate the best treatment options for those patients, but also support the registration of a new paediatric formulation of temozolomide, which is much sought-after for our younger patients.”

Professor Amos Burke, Director of the Cancer Research Clinical Trials Unit concluded: “A liquid formulation of temozolomide is welcomed for younger patients and the use of data from the standard capsule formulation of temozolomide used in BEACON to support registration for a new liquid formulation demonstrates the importance of the collaborative use of results from this practice-changing trial.”