Synlogic Reports Positive Results in Early-Stage Study of Treatment for UCD

November 9, 2017

Rare Daily Staff

Synlogic reported positive top-line clinical data from its early-stage trial of experimental therapeutic SYNB1020 as a treatment for hyperammonemia, an excess of ammonia in the blood in patients with genetic urea cycle disorders or chronic liver disease.

Urea cycle disorder is a genetic condition that is caused by a mutation that results in a deficiency of one of the enzymes that play a role in the urea cycle. These enzymes remove ammonia from the blood. Ammonia, a byproduct of protein metabolism, is converted to urea, which is excreted from the body as urine.

Synlogic is applying synthetic biology to probiotics to develop what it dubs “Synthetic Biotic medicines. SYNB1020 is a first-in-class Synthetic Biotic treatment that is orally delivered and designed to treat hyperammonemia in genetic urea cycle disorders or in chronic liver disease

The company reported that a placebo-controlled single and multiple ascending dose early-stage clinical trial met the primary objectives demonstrating safety and tolerability in healthy volunteers and identified the maximum tolerated dose. It also said the study provided proof of mechanism was demonstrated by a statistically significant dose-dependent effect on the level of a nitrogen endpoint, providing evidence to support its mechanism of action signal in a plasma nitrogen endpoint.

“These data support the hypothesis that SYNB1020 treatment may provide clinical benefit in patients with UCDs or liver disease,” said Aoife Brennan, Synlogic’s chief medical officer. He said the study will inform dose selection for the company’s planned phase 1b/2a study of SYNB1020 in patients, which he expects to begin in the first half of 2018.

November 9, 2017

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