HOUSTON, (October XX, 2023) —Diakonos Oncology Corporation (“Diakonos”), a clinical stage immuno-oncology company, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s unique dendritic cell vaccine, DOC1021. The designation was based on positive preliminary safety and efficacy data from a Phase 1 clinical trial enrolling patients with glioblastoma multiforme (GBM).

FDA Fast Track designation is intended to speed development and review of drugs that show early clinical promise in treating severe or life-threatening conditions. This pivotal designation can help propel Diakonos closer to delivering DOC1021 to GBM patients, of which only 7% survive more than five years.

“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, “Our protocol represents a first for cancer immunotherapy and could be viable for many types of cancers beyond GBM.”

Developed at the Texas Medical Center in Houston, DOC1021 represents a novel approach to fighting cancer that harnesses the body’s natural anti-viral immune response.  By mimicking a viral infection with the patient’s cancer markers, DOC1021 leverages the body’s innate ability to detect and eliminate infected cells.

At the core of DOC1021 is Diakonos’ proprietary “double-loading” technique, which stimulates a previously undiscovered pathway for viral recognition and response. Using a patient’s dendritic cells, a type of white blood cell that detects threats, the unique cancer markers are loaded both internally and externally into the immune cells, which would simultaneously occur in a viral infection. Once the patient’s individualized treatment is prepared, it is administered through three precise injections targeting the deep cervical lymph node chains. This approach results in immune responses that directly target the central nervous system.

This revolutionary approach has shown remarkable outcomes, as the earliest patients have all exceeded survival expectations. DOC1021 also maintains an impressive safety profile. As the treatment enters the final stages of the clinical trial, no serious adverse events have been linked to it. In addition, without the need of genetic modification or artificial stimulation, DOC1021 further stands apart from other cancer immunotherapies.

“Because Phase I clinical trials are generally not statistically powered to demonstrate efficacy, detection of a statistically significant efficacy signal is very promising,” said William Decker, Associate Professor of Immunology at Baylor College of Medicine and inventor of the DOC1021 technology.

Dr. Joseph Georges, FDA Sponsor-Investigator of the DOC1021 phase 1 clinical trial and Assistant Professor of Neurosurgery at the University of Arizona College of Medicine-Phoenix, said, “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. 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.”

The Phase 1 open-label trial of DOC1021 (NCT04552886) is underway at the MD Anderson Cancer Center at Cooper University Health Care in Camden, NJ, and at the University of Texas Health Science Center in Houston. The trial is expected to complete this year.

GBM is the most common and lethal malignant brain tumor with an annual incidence of 3.19 per 100,000 persons in the U.S. About 61% of patients have the unmethylated subtype, which has demonstrated median survival of 15 months compared to 21.7 months for methylated GBM patients when treated with the standard of care.T04552886)