TVI-Brain-1, an immunotherapy being developed by TVAX Biomedical, was given fast track designation by the U.S. Food and Drug Administration (FDA) as a potential treatment of glioblastoma multiforme, a deadly form of brain cancer.
A therapy candidate is named to the fast track program if it might treat serious conditions with unmet clinical need, either because no treatments are currently available or because the potential treatment offers significant benefits over approved alternatives.
This designation is meant to speed the development and review of TVI-Brain-1, facilitating discussions with the FDA and enabling the therapy to qualify for priority review and accelerated approval, provided that certain criteria are met. TVI-Brain-1 has also been named an orphan drug by the FDA.
“We are very pleased to receive Fast Track Designation by the FDA for glioblastoma multiforme (GBM),” Wayne Carter, chief executive officer of TVAX, said in a press release. “Glioblastoma is a devastating disease for which there are limited treatment options.”
TVI-Brain-1 belongs to TVAX’s vaccine-enhanced adoptive T-cell therapy (VACT), an approach to cancer treatment that combines a cancer vaccine with adoptive T-cell therapy.
Initially, patients are vaccinated with their own cancer cells along with an adjuvant to boost the generation of T-cells, immune cells that are able to recognize — but not kill — cancer cells. Then, these T-cells are harvested from the patient’s blood, amplified in the lab, and activated to generate ‘killer’ T-cells. The killer T-cells are returned to the patient via an infusion, so they can seek out and destroy tumors.
The candidate therapy has been tested in Phase 1/2 (NCT01081223) and Phase 2 (NCT01290692) trials in patients with advanced brain cancers, including glioblastoma multiforme, who failed to respond to surgery, radiotherapy, and chemotherapy.
A significant number of patients were reported to experience meaningful reductions in tumor volume (23%) after treatment. Their median overall survival was also extended by over six months compared with historical controls.
TVI-Brain-1’s use was well tolerated, without signs of treatment-related serious adverse events. The most common side effects were linked to inflammatory responses resulting in flu-like symptoms — fever, chills, headache, nausea — that often resolved within one to two days.
TVAX is planning to open a Phase 2b trial testing TVI-Brain-1 in people newly diagnosed with glioblastoma multiforme this year. People likely to be enrolled are expected to have healthier immune systems — not compromised by chemotherapy — and relatively smaller tumors.
