Cancer Vaccine BN-Brachyury Granted Orphan Drug Designation

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to novel 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.

Chordoma is diagnosed in just 1 in 1,000,000 people per year.

That means that about 300 patients are diagnosed with chordoma each year in the United States.

Nuclear Brachyury (T) 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 2 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, Bavarian Nordic A/S initiated an open-label Phase 1 trial to evaluate the safety and tolerability of the BN-Brachyury vaccine. This trial is currently enrolling up to 10 patients with metastatic or unresectable, locally advanced malignant solid tumors.

The primary endpoint of this small 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.

"We are very happy to obtain orphan status for our chordoma program, given the severe lack of effective therapies for this population of patients. We plan to initiate our phase 2 study in the fall of this year and to rapidly progress this program through proof of concept," said Paul Chaplin, President, and CEO of Bavarian Nordic.

The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Bavarian Nordic is a biotechnology company focused on the development of innovative and safe therapies against cancer and infectious diseases. Using our live virus vaccine platform technology, MVA-BN®, we have created a diverse portfolio of proprietary and partnered product candidates intended to improve the health and quality of life for children and adults. For more information visit www.bavarian-nordic.com