Mustang Bio stock soars after FDA grants orphan drug designation

Published 07/07/2025, 13:54
© Reuters.

Investing.com -- Mustang Bio Inc (NASDAQ:MBIO) stock surged over 78% after the U.S. Food and Drug Administration granted orphan drug designation to its MB-101 treatment for recurrent diffuse and anaplastic astrocytoma and glioblastoma, rare forms of brain cancer.

The designation provides Mustang Bio with several benefits, including tax credits for clinical trials and seven years of market exclusivity if the treatment receives regulatory approval. The FDA’s designation covers a broader indication than what was originally proposed by the company.

MB-101 consists of IL13Ra2-targeted CAR T-cells designed specifically to target these aggressive brain cancers. In an ongoing Phase 1 trial published in Nature Medicine, the treatment showed promising results with 50% of patients achieving stable disease or better outcomes. Notably, two patients achieved complete responses lasting 7.5 and 66+ months respectively.

The study evaluated 57 patients total, with the two complete responses occurring in a cohort of three patients who had the highest levels of intratumoral CD3+ T cells before treatment.

Mustang Bio is also developing MB-109, a combination therapy using MB-101 with MB-108, an oncolytic virus treatment for malignant glioma that previously received orphan drug designation. This combination approach uses MB-108 to modify the tumor microenvironment to potentially enhance MB-101’s effectiveness.

The company noted that further development of the MB-109 program depends on securing additional funding or establishing a strategic partnership. Mustang licenses MB-101 from City of Hope and MB-108 from Nationwide Children’s Hospital.

Phase 1 clinical trials for both treatments continue enrolling patients at City of Hope and The University of Alabama at Birmingham.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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