The US Food and Drug Administration (FDA) has granted orphan drug designation to Diakonos’ dendritic cell vaccine DOC1021 for malignant glioma.

If the therapy is approved, the US-based immuno-oncology company is eligible for a potential seven years of market exclusivity in newly diagnosed or refractory glioblastoma multiforme, according to a 2 January press release. The orphan drug status also provides tax credits for US-based clinical trials.

In the same press release, Diakonos stated it had completed enrolment in a Phase I trial investigatingDOC1021.

The single-arm, first-in-human study (NCT04552886) is evaluating the safety of the personalised vaccine administered to patients with glioblastoma who have completed standard-of-care treatments including chemotherapy and radiation therapy. Progression-free survival and overall survival will be evaluated as secondary outcomes.

Diakonos has administered four dose levels of its dendritic cell vaccine to a total of 16 patients in the trial. The company reported a good safety profile of DOC1021, with no attributable serious adverse events.

Highlighting efficacy signals, Diakonos added that 13 of the patients remain alive. The first patient was enrolled in October 2021. Around 5% of patients with glioblastoma multiforme currently survive beyond five years, while the average survival time is 12-18 months.

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Diakonos has previously received a fast track designation from the FDA for the vaccine in October 2023. This designation speeds up the development and review of drugs showing early clinical promise for severe or life-threatening conditions.

Diakonos’ chief regulatory officer Ian Bellayr said: “Receiving the orphan drug designation from the FDA is a valuable addition to our existing fast track designation as it indicates that the cell therapy may be more broadly applicable to other oncological indications.”

Diakonos’ CEO Mike Wicks added that the company’s focus is now shifting towards manufacturing optimisation and enrolment of additional sites for the upcoming Phase II trial.

In October 2023, Diakonos partnered with contract development and manufacturing organisation Cellipont Bioservices for the process development and cGMP manufacturing of DOC1021.

DOC1021 is made from a patient’s dendritic cells and a sample of their tumour. This allows the therapy to target the complete cancer antigen profile while leaving the patient’s immune cells alone.

Dendritic cells are responsible for initiating antigen-specific immune responses. Diakonos said its research has shown a strong killer T cell response and the formation of immunological memory to prevent cancer recurrence.

Diakonos has another dendritic cell vaccine candidate for the treatment of pancreatic cancer, which is currently in a Phase I trial (NCT04157127).

In November 2023, the FDA granted orphan drug designation to MAIA’s cancer telomere-targeting agent THIO for glioblastoma multiforme patients.