Therapy for aggressive brain cancer receives FDA clearance to start phase 1
US biotech company Adaptin Bio has emerged from stealth with the announcement that the FDA has cleared an Investigational New Drug application (IND) for its program APTN-101, a therapy targeting glioblastoma, also known as WHO classified grade 4 malignant glioma.
In the US, approximately 15,000 people are diagnosed with glioblastoma every year. It is the most aggressive form of brain cancer with a very poor prognosis. Fewer than 5% of glioblastoma patients survive five years after diagnosis. Current treatment options are limited and include chemotherapy, radiotherapy, and surgery. However, due to the aggressive and infiltrating nature of the cancer, disease recurrence is common as the tumor cells often evade treatment.
Adaptin Bio’s APTN-101 aims to address this challenge by crossing the blood-brain barrier and targeting aggressive brain tumor cells directly. The therapeutic is based on the company’s proprietary Brain Bispecific T cell Engager (BRiTE) platform, which couples engineered T cells with bispecific antibodies, improving the transport of these antibodies to the brain and other tissues.
In the case of APTN-101, the bispecific antibody is designed to simultaneously target a mutant protein called EGFRvIII that is commonly found on glioblastoma cells, and the CD3 receptor on T cells. This way, the T cells are activated and directed to the tumor, where they can target and kill the glioblastoma cells directly.
Commenting on Adaptin Bio’s recent announcement, Mustafa Khasraw, professor of neurosurgery, medicine, integrative immunobiology, cancer biology, and pharmacology at Duke University in North Carolina, where the BRiTE platform was developed, said: “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.”
Preclinical studies revealed that APTN-101 was greatly distributed in the brain – meaning it had successfully passed the blood-brain barrier – and was effective in eliminating malignant glioma tumors across several preclinical models.
“We’re thrilled to initiate clinical trials with APTN-101 following FDA clearance of our first-in-human trial,” said Michael Roberts, 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. We are committed to advancing this novel therapy as a new potential therapy for glioblastoma patients who desperately need new therapies.”
The phase 1 clinical study will look at the safety and efficacy of APTN-101 in patients with grade 4 malignant glioma. Adaptin Bio has yet to announce the starting date for the trial.
