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May 12, 2014
 |  BioCentury  |  Emerging Company Profile

Pharmasum: Down with AD

Pharmasum targeting beta amyloid, tau pathways of Alzheimer's in Down syndrome

Pharmasum Therapeutics A/S is looking to treat the large number of Down syndrome patients who develop Alzheimer's disease-like pathology. The company's first-in-class inhibitor could slow cognitive decline in Down syndrome by targeting a kinase that regulates two key pathogenic pathways in AD.

Cancer company Lytix Biopharma A/S spun out Pharmasum this year after screening studies "serendipitously identified small molecules that were surprisingly active and selective for protein kinases that have been implicated in neurological diseases rather than cancer," said Anders Fugelli, CEO of Pharmasum and former head of business development at Lytix.

The lead program Pharmasum received from Lytix, PST-900, inhibits dual-specificity tyrosine-(Y)-phosphorylation regulated kinase 1A (DYRK1A), a protein involved in neuronal development, maturation and aging. DYRK1A also regulates two pathways involved in AD pathogenesis: the formation of beta amyloid plaques and the phosphorylation of microtubule-associated protein tau (MAPT; tau; FTDP-17) that induces formation of neurofibrillary tangles.

Over the past five years, studies have linked up-regulation of DYRK1A in the hippocampus to the development...

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