FDA Grants Orphan Drug Designation to BN-Brachyury for the Treatment of Chordoma

On May 2, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to the cancer vaccine BN-Brachyury for the treatment of chordoma.

Chordoma is a rare type of cancer that develops along the spine, with presentation occurring at one of three main sites: sacrum, mobile spine, or the clivus (skull base). It shares many characteristics with sarcomas, but is often characterized as a bone tumor. Chordomas are complicated tumors to treat, due to the proximity to and/or involvement with critical structures such as the brainstem, spinal cord, and important nerves and arteries.

Nuclear Brachyury expression has emerged as a sensitive and fairly specific diagnostic marker for chordoma. A growing body of literature suggests that it contributes significantly to chordoma pathogenesis.

A phase II study in patients with metastatic chordoma will initiate in the second half of 2018, enrolling up to 25 patients, with a goal of increasing overall response rates for patients receiving the BN-Brachyury vaccine in combination with radiation therapy. In early 2018, an open-label phase I trial to evaluate the safety and tolerability of the BN-Brachyury vaccine was initiated. This trial is currently enrolling up to 10 patients with metastatic or unresectable, locally advanced malignant solid tumors. The primary endpoint of the study is safety and tolerability, and secondary endpoints include immunologic responses, as measured by an increase in brachyury-specific T cells and other tumor-associated antigens.

Chordoma is diagnosed in about 300 patients each year in the United States, and about 700 in all of Europe. At any given time, it is estimated that fewer than 1 in 100,000 people are living with chordoma.

About BN-Brachyury

BN-Brachyury is a novel prime-boost cancer immunotherapy candidate, developed in collaboration with the National Cancer Institute (NCI). The product candidate consists of a prime (MVA-BN) and a booster dose (fowlpox or FPV), which have been modified to express brachyury and to encode three costimulatory molecules, known as TRICOM. Brachyury is a tumor-associated antigen that is overexpressed in major solid tumor indications, as well as several rare, ultra-orphan cancer indications, and is reported to play a key role in the metastasis and progression of tumors. Tumors that overexpress brachyury are believed to be highly resistant to standard therapies, including radiation and chemotherapy, and are associated with decreased survival rates.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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