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Cambridge startup Acer Therapeutics is developing the first therapy for a rare inherited condition called Maple Syrup Urine Disease. Known for causing neurological and social impairment, MSUD gets its unique name from the urine’s distinctive smell of maple syrup – all thanks to a defect in an individual’s amino acid metabolism.

The company just raised $2.1 million, according to an SEC filing. The startup’s business model is to repurpose and reformulate existing therapeutics, such as sodium phenylacetate and sodium phenlybutyrate, that work in related conditions.

The technology is licensed out of Baylor University. The school’s actually leading the startup’s double-blinded Phase 3 clinical trial, which is currently enrolling patients.

MSUD patients carry a genetic defect that prevents branched chain amino acids from being broken down. These, and their corresponding ketoacids, build up abnormally in the system – lending the maple syrup smell to the urine. This also triggers neurological impairment, coma, even death.

The disease falls solidly in the orphan bucket – there are about 900 patients with MSUD in the U.S., and 4,000 around the world. It has a worldwide incidence of one in 185,000 births, though interestingly, it’s reported in one in 350 births among the Old Order Mennonite population.

Right now, there isn’t an effective treatment for MSUD, outside of dietary restriction. Acer’s lead aim is to test out phenylbutyrate, because the drug’s previously been found to reduce branched chain amino acids when consumed. Acer’s found success preclinically for this drug, as well as for sodium phenylacetate/benzoate.

Oh, and interesting: The startup got its name from the genus of the maple tree. Acer.


Original article from http://medcitynews.com/2015/04/cambridge-startup-raises-2-1m-maple-syrup-urine-disease/. By Meghana Keshavan. Meghana has spent the last year-and-a-half writing about biotech and healthcare for the San Diego Business Journal. Previously, she's worked for Reuters, Crain’s Detroit Business, the Detroit Free Press and WDET, a Detroit-based National Public Radio affiliate. Meghana studied biochemistry and molecular biology at Penn State University and spent five years as a self-described "research peon in a schizophrenia genetics lab."





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