12:00 AM
Mar 14, 2016
 |  BioCentury  |  Emerging Company Profile

AMO's CNS ammunition

AMO is repurposing compounds to treat CNS deficits in fragile X and DM1

AMO Pharma Ltd. is in-licensing therapies for rare genetic diseases with no treatment options, starting with two potentially disease-modifying candidates for fragile X syndrome and myotonic dystrophy type 1.

The company's first candidate is AMO-01, a farnesylated dibenzodiazepinone in development for fragile X. AMO-01 targets the peripheral benzodiazepine receptor (TSPO; PBR), expressed on activated astrocytes, and inhibits the Ras-ERK pathway.

AMO licensed AMO-01 from Thallion Pharmaceuticals Inc. (now part of Bellus Health Inc.). Thallion had discontinued a Phase II trial of AMO-01 in glioblastoma multiforme (GBM) due to lack of efficacy.

In fragile X, the compound could reverse underlying synaptic defects. The disease is characterized by impaired neural and cognitive development caused by transcriptional silencing of fragile X mental retardation 1 (FMR1), the gene encoding the fragile X mental retardation protein (FMRP). The absence of FMRP leads to increased activation of the Ras-ERK pathway, leading to defective synaptic pruning and excessive synaptic connections.

CEO and CSO Michael Snape said unpublished data show AMO-01 induced normal synaptic pruning and affected all symptom...

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