Acer Therapeutics Receives Patents in the U.S. and EU for the use of Sodium Phenylbutyrate (NaPB) and Prodrugs for the Potential to Treat Maple Syrup Urine Disease (MSUD)

The United States Patent and Trademark Office (USPTO) and the European Patent Office (EPO) have issued U.S. Patent 9,078,865 and EU patent 2,456,304, respectively, covering ACER-001, a proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate (NaPB), for treating Maple Syrup Urine Disease (MSUD). MSUD is a rare, devastating genetic disease which prevents the proper metabolism of three essential, branched-chain amino acids (BCAAs) – leucine, isoleucine and valine. Acer filed the recently issued patents in 2010, which would expire in 2030 without extensions.

There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment. Approximately 1,000 people suffer from MSUD in the U.S. and 3,000 are affected worldwide.

ACER-001 was granted orphan drug designation by the FDA in 2014. This designation, along with the recently issued patents, strengthens the exclusive position of Acer in advancing ACER-001 as the first potential therapy for the treatment of MSUD.