BREN-02 is expected to be administered in clinical trials in ALS patients in the second half of 2021, subject to preclinical toxicity results and regulatory review. Preclinical studies have shown that the homeoprotein hEN1 is essential for the survival and maintenance of spinal cord alpha motor neurons that innervate muscles throughout the body.
The FDA grants orphan drug status to encourage the development of therapies to treat, prevent or diagnose diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation in the United States recognizes the therapeutic potential of BREN02 in ALS and allows BrainEver to benefit from specific measures and advantages, including a seven-year marketing exclusivity period if hEN1 is approved for the treatment of ALS.
