July 22, 2008
FRAXA Research Foundation and a UK-based company, Neuropharm, have been working together to explore the potential of fenobam to treat Fragile X and possibly autism. Fenobam is a compound which reduces mGluR5 activity, which is known to be overactive in Fragile X. Neuropharm received Orphan Drug Designation in the US for fenobam to treat
Fragile X Syndrome after acquiring rights to relevant data on the compound from
FRAXA.
Now - just in time for National Fragile X Awareness Day - the results of a Phase IIa trial in Fragile X patients are available - and encouraging! This trial was conducted by Professor Randi Hagerman of the UC Davis
MIND Institute and Professor Elizabeth Berry-Kravis of the RUSH University Medical Center.
Highlights of the fenobam trial:
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This was a single dose, open label study of fenobam in 6 male and 6 female adults with
Fragile X Syndrome.
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The study investigated the tolerability and pharmacokinetics of fenobam in this patient
group. The study also investigated the effect of fenobam on additional efficacy related outcome
measures. These measures had the potential to measure efficacy after administration of a
single dose.
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The compound was well tolerated: there were no significant adverse reactions.
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Four of 6 males and 2 of 6 females were responders according to pre-defined criteria of
efficacy
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The investigators concluded that the favorable safety profile and the clinical effects
noted in this study support implementation of further controlled trials of NPL-2009 in
adults with Fragile X Syndrome.
Robert Mansfield, Neuropharm’s CEO, commented: "This represents a significant step
forward in the Fragile X arena and we acknowledge this achievement by Professors
Hagerman and Berry-Kravis. We are delighted to be working with the principal investigators
and with the team at FRAXA on this exciting Orphan Drug project."
Future trials of fenobam and other targeted treatments for Fragile X will be announced at FRAXA.org and at ClinicalTrials.gov
Neuropharm Limited, a subsidiary of Neuropharm Group plc, is a UK based speciality pharmaceutical group focused on the development of products for the treatment of disorders of the central nervous system. FRAXA Research Foundation is a US-based parent-run nonprofit organization which funds research aimed at finding effective treatments and a cure for Fragile X.
