SOURCE: NeuroInvestment


December 01, 2010 13:21 ET

Fragile X Therapeutics Programs Reviewed by NeuroInvestment

CARDIFF, CA--(Marketwire - December 1, 2010) - NI Research has released the December issue of NeuroInvestment, which features a comprehensive review of therapeutics for the treatment of Fragile X.

Fragile X has become a focus for a growing number of pharmaceutical companies as they have considered the potential value of addressing an orphan disorder which is a significant neurodevelopmental disorder in its own right, the single most common form of inherited mental disability/retardation. Successful disease-modification in Fragile X may also be the gateway to disease-modifying treatments for autism, and perhaps even schizophrenia.

The phenotype includes a constellation of cognitive, emotional, and physical features, including prototypical physical/facial characteristics. The behavioral/functional expression of Fragile X, which can range from relatively mild to very severe, can include autism (autism emerges in 25-30% of the Fragile X population: the largest genotypic cohort associated with autism, estimated to constitute about 5% of the autism population), aggressiveness (in about 30%), impoverished language skill development, and in some cases, seizure risk or motor ataxia.

The Fragile X mutation leads to the underproduction of FMRP, an essential mediator of synaptic protein synthesis, leading to defects in both structure and cognitive function. The major approaches to Fragile X, beyond the crude attempts at neuroleptic symptom control currently available, include mGluR5 antagonism (Seaside Therapeutics, Novartis, Roche), GABA-B antagonism (Seaside Therapeutics), and PAK inhibition (Afraxis). Other potential strategies include neurotrophic/protective drugs (Neuren, Cortex Pharmaceuticals) and oxytocin (Cypress Bioscience). Hint of concept has been established in pilot studies conducted by Novartis and Seaside Therapeutics.

The December issue also includes:

1) The Picomolar Solution: What happens when a billion dollars is distributed with such a low barrier to access that companies who are moribund or even out of business received grants? The effect of the stimulus is so diluted as to be virtually useless. The 5.5% of the Therapeutics Grants program that went to CNS programs is detailed and discussed.

2) Commentary on Valeant's crude, regressive, and complete dismantling of the Biovail CNS development program.

3) A Special Report on the current state of the CNS drug development area, with specific recommendations regarding changes the industry needs to make to bring CNS back into the forefront, and to reverse the current exodus of resources and credibility from the CNS sector. The factors considered include the degree of optimal information-sharing and collaboration between companies; replacing program prioritization based on often flimsy theoretical assumptions with an 'Agnostic Audition' approach to establishing HOC and POC; and detailing the kinds of core clinical questions that, if just one is answered in the affirmative, will bring CNS back into the pharmaceutical spotlight.

4) A succinct assessment of Afraxis, focused on Fragile X and autism.

NeuroInvestment is the independent, monthly review of the neurotherapeutics area. Its reputation has been built on over fifteen years of objective, insightful, and critical coverage of the neurotherapeutics field. A one-year, five-user subscription is $2100, email or hardcopy. Add $250 for dual delivery, add $50 for airmail delivery outside North America. A three month email trial is US$700. The December issue is available as a single purchase, for US$300.

The November issue of NeuroInvestment reviewed programs for the treatment of neuropathic pain, and included an assessment of Convergence Pharmaceuticals.

NI Research is the leading publisher of independent research on the neurotherapeutics industry. NI Research has published NeuroInvestment since 1995, the Private CNS Company Review since 2003, and CNS Disorders/Therapeutics since 2007. NI Research also provides strategic consultation services. NI Research has developed an unmatched information base regarding both publicly and privately-held neuro-oriented companies.

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