WORCESTER, Mass. - Mustang Bio , Inc. (NASDAQ:MBIO), a clinical-stage biopharmaceutical company, has announced Phase 1 clinical trial results for MB-101, its CAR-T cell therapy for recurrent and refractory malignant glioma. Published in Nature Medicine, the study shows MB-101, developed by City of Hope and licensed to Mustang, was well-tolerated, with 50% of patients achieving stable disease or better.
The trial, which is the largest to date for CAR-T therapy in solid tumors, reported on 65 patients, primarily with glioblastoma. Patients treated with dual intratumoral and intraventricular delivery, along with an optimized manufacturing process, had a median overall survival of 10.2 months, surpassing the expected six months for recurrent glioblastoma. The overall median survival was eight months.
The therapy was administered directly into the brain tumor and cerebrospinal fluid to bypass the blood-brain barrier, a significant challenge in treating brain cancer. The study found that patients with higher pre-treatment tumor T-cell levels, indicative of a "hot" tumor microenvironment, showed a significant survival benefit.
According to Dr. Christine Brown from City of Hope, the results indicate a significant step forward in treating this aggressive tumor type with limited treatment options. MB-101 was delivered in doses up to 200 x 10^6 CAR-T cells per infusion, showing no dose-limiting toxicities.
Common toxicities potentially related to the treatment were fatigue, headache, and hypertension, with some patients experiencing transient grade 4 cerebral edema and grade 3 encephalopathy or ataxia.
Mustang's President and CEO, Manuel Litchman, M.D., highlighted the complete responses achieved in two patients, lasting 7.5 and 66+ months, respectively. He expressed optimism for the upcoming combination clinical trial of MB-101 with MB-108, an oncolytic virus, to improve treatment outcomes.
The Phase 1 study's primary endpoints focused on safety and feasibility, with secondary endpoints assessing cytokine dynamics, CAR-T cell persistence, and clinical outcomes. No high-grade cytokine release syndrome or immune effector cell-mediated neurotoxicity adverse events were observed.
This report is based on a press release statement from Mustang Bio, Inc. Dr. Brown has a financial interest in Mustang and has previously been a paid consultant for the company. Additional details on the study can be found on ClinicalTrials.gov under identifier NCT02208362.
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