• Phase I trials indicated BN-Brachyury is well tolerated
• Initiating phase 2 study in patients with metastatic chordoma
• Also has the potential to treat: triple negative breast cancer and non-small cell lung cancer which are known to have high expression levels of brachyury

Bavarian Nordic A/S (OMX: BAVA, OTC: BVNRY) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the Company's 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. 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, the Company 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 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.

"We are very happy to obtain orphan status for our chordoma program, given the severe lack of effective therapies for this population of patients. Chordoma is a unique tumor where the brachyury protein is universally overexpressed; which presents us with an exceptional opportunity to use our vaccine in a optimally targeted manner. 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.

A dose escalation Phase 1 clinical trial (NCT02179515) that included 38 participants with advanced chordoma aimed assess the drug’s safety and identify brachyury-specific T-cell responses. Three under-the-skin vaccine dose levels were evaluated.

BN Brachyury was well tolerated with no dose-limiting toxicities observed. Researchers saw T-cell responses at all dose levels in most participants, and recorded no serious side effects related to the treatment.