DOC1021 Gains FDA Fast Track Designation in Glioblastoma Multiforme

• DOC1021 is a dendritic cell vaccine under investigation as a potential therapeutic option for patients with glioblastoma multiforme.

• The treatment is currently being evaluated in a single-arm, open-label, first-in-human phase 1 trial (NCT04552886), which has an estimated study completion date of December 2025.

• Phase 1 safety and efficacy findings support this regulatory decision.

The FDA granted a fast track designation to DOC1021, a dendritic cell vaccine, as a potential treatment option for patients with glioblastoma multiforme, according to Diakonos Oncology Corporation.¹

“The FDA’s decision acknowledges the potential of this new treatment approach for a very challenging disease,” said Mike Wicks, chief executive officer of Diakonos, in a press release. “Our protocol represents a first for cancer immunotherapy and could be viable for many types of cancers beyond glioblastoma multiforme.”

Human Brain: © blueringmedia- stock.adobe.com

DOC1021 is a multi-step process which uses a patient’s natural immune response, and activates an antiviral immune response to target and eliminate cancer cells.² The treatment was developed at the Texas Medical Center in Houston, and uses a “double-loading” technique which stimulates a novel viral recognition and response pathway.

The basis of this regulatory decision is supported by preliminary safety and efficacy findings from a phase 1 trial (NCT04552886).

“The vaccine's mechanism of action and its unique route of administration showcase the potential of harnessing the body's immune system to combat glioblastoma,” said Joseph Georges, DO, PhD, assistant professor of neurosurgery at the University of Arizona College of Medicine in Phoenix, in a press release.¹ “Historically, glioblastoma outcomes have been notoriously challenging to improve upon. From a clinical and scientific standpoint, the results we are observing with DOC1021 are encouraging.”

Currently, a single-arm, open-label, first-in-human phase 1 trial is investigating the safety and feasibility of DOC1021 among patients with glioblastoma and plans to explore the primary end point of safety and potential toxicity of DOC1021, as well as the secondary end points of overall survival and progression-free survival.³

The study plans to enroll 9 to 24 adult patients with glioblastoma who have undergone neurosurgical tumor resection and in whom a neuropathological diagnosis has been established.² Those eligible for enrollment in the trial are patients with potentially resectable glioblastoma, and those deemed good candidates for adjuvant chemotherapy and radiation therapy. This includes patients with tumors that are suitable for gross total resection or partial resection. Additionally, patients are required to have an ECOG performance status of 2 or lower, and adequate liver, kidney, immune, and bone marrow function.

At the start of the study, between 3 and 6 patients will be treated with a starting dose of 3.5 x 10⁶ of DOC1021. If the dose is associated with unacceptable adverse effects (AEs), patient enrollment at this dose would be completed, and 3 to 6 patients will be given the vaccine at a dose of 1.75 x 10⁶ cells in the de-escalation cohort. If the starting dose does not correlate with unacceptable AEs, 3 to 6 patients in a dose-escalation cohort will receive a dose of 7.0 x 106 cells. If there are no unacceptable AEs associated with the vaccine at 7.0 x 10⁶ cells, a second dose-escalation cohort consisting of 3 to 6 patients will be treated with a dose of DOC1021 at 1.4 x 10⁷ cells.

“Because phase 1 clinical trials are generally not statistically powered to demonstrate efficacy, detection of a statistically significant efficacy signal is very promising,” William Decker, PhD, an associate professor of immunology at Baylor College of Medicine and the inventor of the DOC1021 technology, said in the news release.¹

The trial is ongoing and recruiting patients at the MD Anderson Cancer Center at Cooper University Health Care in Camden, New Jersey, and at the University of Texas Health Science Center.² The study has an estimated completion date of December 31, 2025.

REFERENCES

3. Diakonos Oncology awarded FDA fast track designation for innovative dendritic cell vaccine for glioblastoma. News Release. Diakonos Oncology. October 17, 2023. Accessed October 17, 2023. https://tinyurl.com/mr3x476b

2. Diakonos expanded access. Diakonos Oncology. Accessed October 17, 2023. https://tinyurl.com/kvyc45mv

3. Dendritic cell vaccination with standard postoperative chemoradiation for the treatment of adult glioblastoma. ClinicalTrials.gov. Updated June 28, 2023. Accessed October 17, 2023. https://tinyurl.com/28p4w7v6