Acer Therapeutics Licenses Exclusive Worldwide Rights from Baylor College of Medicine for Development of Sodium Phenylbutyrate for Maple Syrup Urine Disease

In April 2014, Acer Therapeutics Inc. entered into an exclusive worldwide license agreement with Baylor College of Medicine to develop and commercialize formulations and prodrugs of sodium phenylbutyrate for the treatment of 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. 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.

“The research data generated by the team at Baylor College of Medicine supports  phenylbutyrate, when used in conjunction with a BCAA-restricted diet, as a promising therapy to lowering levels of BCAAs – and in particular leucine – for patients affected by MSUD,” said Chris Schelling, COO and Founder of Acer Therapeutics. “We are looking forward to advancing our proprietary taste-masked, immediate-release formulation of sodium phenylbutyrate, ACER-001, through clinical studies and potentially bring the first therapeutic option to patients with this serious disease.”