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Roche signs research deal on autism spectrum disorders

Published on 19/06/12 at 12:14pm
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Roche has signed an agreement with Seaside Therapeutics to develop new treatments for autism and other neurodevelopmental conditions such as Asperger’s syndrome.

The alliance will focus on targeting the molecular basis of a rare genetic disease, fragile X syndrome (FXS), and autism spectrum disorders (ASD). No financial details have been disclosed.

“Recent discoveries in genetics have shed light on the biological underpinnings of these conditions thus providing a basis for mechanistic drug discovery,” said Luca Santarelli, global head of Roche Neuroscience.

FXS’s symptoms resemble ASD, and their underlying mechanisms may be similar, the companies believe.

The most commonly inherited form of autism involves the gene encoding fragile X mental retardation protein (FMRP): loss of this function disrupts signalling between neurons, leading to brain abnormalities.

FMRP is balanced by mGluR5, a brain receptor involved in learning and memory, and early trial data suggests children with FXS could be helped by drugs that inhibit mGluR5 activity.

With this in mind, Seaside will licence patents covering the use of mGluR5 antagonists in these therapy areas exclusively to Roche, who will then lead development and commercialisation.

Roche’s own mGluR5 drug candidate, RG7090, is currently enrolling patients in a Phase II trial in FXS and the manufacturer is looking at  biomarkers to identify patients likely to benefit.

Meanwhile Seaside is about to start a Phase III trial in FXS and Phase IIb trial in ASD with STX209, an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist.

GABA-B receptors can help control the activation of glutamate receptors and ratios of excitatory to inhibitory neurotransmission in the brain - high levels of which are thought to be part of some neurodevelopmental disorders.

With STX209 Seaside has already completed a randomised, blinded, placebo-controlled Phase II trial in patients with FXS and an open-label Phase IIa exploratory trial in ASD, and the deal with Roche gives it the money to complete late-stage clinical development.

Seaside will retain exclusive rights to patents covering the use of GABA-B agonists for the treatment of FXS and ASD, although Roche is able to exercise some options to commercialise STX209.

Seaside chief executive Randy Carpenter said the drug “has the potential to change the treatment paradigm for fragile X and autism and thereby help patients and their families achieve an improved quality of life”.

Adam Hill

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