FDA OKed New Drug Application for Novel Glioblastoma Treatment

The novel drug, APTN-101, can now be studied in certain patients with glioblastoma.

The Food and Drug Administration (FDA) has cleared an investigational new drug application for the novel treatment, APTN-101, for glioblastoma, a form of brain cancer.

Glioblastoma is a type of fast-growing and invasive cancer that grows in the brain or spinal cord, according to the Mayo Clinic. Of note, this type of cancer may occur at any age, but is more common in older adults. Currently, there is no cure for glioblastoma, although treatments work to slow the growth of cancer and reduce symptoms.

Following this clearance, patients with glioblastoma will receive APTN-101 and will be evaluated in an upcoming phase 1 study, according to a news release from Adaptin Bio, the manufacturer of APTN-101. Specifically, researchers will be identifying the safety and efficacy of APTN-101 in patients with grade 4 glioblastoma.

APTN-101 is a type of brain bispecific T-cell engager (BRiTE), which targets the EGFRvlll, a type of protein that is associated with aggressive brain tumors, the news release stated. The technology of this type of drug is able to manipulate immune cells, which deliver the treatment directly to the brain tumor, as explained in the release. Based on preclinical trials, the novel treatment has shown significant benefits, according to the release. 

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"The FDA clearance is a significant achievement for our collaboration with Adaptin. Based on results from preclinical models in orthotopic malignant glioma, we are excited to begin this clinical trial to evaluate the safety and efficacy of APTN-101," said Dr. Mustafa Khasraw, professor of neurosurgery, medicine, integrative immunobiology, cancer biology and pharmacology at Duke University in North Carolina. "APTN-101’s ability to cross the blood-brain barrier and target glioma cells directly offers a promising new approach."

Standard treatment for patients with glioblastoma includes surgery before they receive radiation every day for approximately six and a half weeks along with oral chemotherapy, according to Johns Hopkins Medicine. After, patients typically are treated with a six-month regimen of oral chemotherapy that is taken for five days each month.

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"We’re thrilled to initiate clinical trials with APTN-101 following FDA clearance of our first-in-human trial," said Michael J. Roberts, the CEO of Adaptin Bio. "Our proprietary BRiTE technology harnesses the immune system’s remarkable ability to target and deliver therapeutics to specific tissues, including the brain, potentially revolutionizing treatment for difficult-to-treat cancers. APTN-101 validates the BRiTE platform and its ability to enhance transfer of therapeutics into the brain."

In 2024, there will be an estimated 25,400 new cases of glioblastoma in the U.S., which makes up 1.3% of all new cases of cancer, according to the National Cancer Institute.

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