FDA Breakthrough Designation and IDE Approval for Insightec’s NSCLC Brain Mets Study

By News Release


The US FDA has granted approval of two pivotal investigational device exemptions (IDE) for the Exablate Neuro system from Insightec. One approval is for primary non-small cell lung cancer (NSCLC) that has metastasized to the brain in conjunction with standard-of-care Keytruda, and the other is for enhanced efficacy of liquid biopsy for recurrence monitoring of patients with primary brain cancer. The FDA has also granted "Breakthrough Device" designation for the NSCLC treatment, helping to expedite its development and review.

Insightec plans to launch the LIMITLESS study (NSCLC) to assess the clinical benefit of using noninvasive, low-intensity focused ultrasound combined with systemic immunotherapy in the treatment of patients with brain metastases from lung cancer.

"The use of low-intensity focused ultrasound (LIFU) with microbubbles to open up the blood brain barrier (BBB) is an exciting advance in neuro-oncology that holds promise to become a paradigm shift in the treatment of patients with brain tumors," said study Principal Investigator Dr. Manmeet Ahluwalia, MD, MBA, Chief of Solid Tumor Medical Oncology, Deputy Director and Chief Scientific Officer at Miami Cancer Institute, part of Baptist Health South Florida. "The unique synergy of LIFU BBB opening for improved drug delivery, neoantigen release and immune priming is a potential game-changer in our field that will help us build on current treatment options available for these patients."

Liquid biopsy is a new approach for noninvasive cancer subtyping, treatment selection, monitoring residual disease, early identification of treatment responders versus non-responders, and assessment of tumor progression versus pseudoprogression.

"Despite remarkable progress in other cancers, liquid biopsy has had limited success in brain tumors due to the presence of the blood brain barrier, which limits signal detection in the bloodstream," said Dr. Achal Singh Achrol, MD, FAANS, Chief Medical Officer of Insightec.  "Preclinical and early human studies have demonstrated that low-intensity focused ultrasound (LIFU) can transiently increase the permeability of the blood brain barrier and allow passage of region-specific biomarkers to enable noninvasive liquid biopsy in brain tumors.  This pivotal clinical trial will investigate for the first time the clinical benefit to this approach as a novel alternative to invasive neurosurgical biopsies."

"Insightec continues to collaborate with leading researchers to advance the use of acoustic therapy in the brain," said Maurice R. Ferré, MD, Insightec CEO and Chairman of the Board. "This work focuses on safely opening the blood brain barrier using low frequency acoustic energy and has the potential to transform current therapeutic and diagnostic approaches for conditions like brain cancer. Our ultimate goal is, as always, to positively impact patient lives."