BioWorld International Correspondent
LONDON - Neuropharm Group plc secured the support of the U.S. patients' group Autism Speaks in the staging of a Phase III trial of NPL-2008, a formulation of Prozac (fluoxetine) in treating autism spectrum disorder.
The agreement gives Neuropharm access to the charity's clinical trials network of pediatric centers and to expert clinicians. The randomized, double-blind, placebo-controlled trial, which aims to show a reduction in the core symptomology of autism, will recruit more than 100 children ages 5 to 17, across 12 centers.
Robert Mansfield, CEO of Neuropharm, told BioWorld International, "It's a big advantage getting this support. Autism Speaks has a very well-established network in an area where not many drugs have been trialled, so it's very useful to have them as collaborators."
NPL-2008 is a melt-in-the-mouth formulation of fluoxetine, a selective serotonin re-uptake inhibitor, which is one of the most widely prescribed central nervous system drugs. Neuropharm licensed the right to use the drug in treating autism from Mount Sinai School of Medicine in New York, where a Phase II and Phase III trial were carried out with funding from the FDA's Orphan Drug Office.
The trials did use the melt-in-the-mouth formulation. However, the FDA has told Neuropharm it needs only to show bioequivalence. Mansfield said one of the main objectives of the new trial is to allow clinicians to gain experience in titrating the drug to reach the most appropriate dose for each subject.
A range of publicly funded studies that have been consolidated by the research at Mount Sinai, shows serotonin levels in the brains of autism sufferers are far lower than normal.
Treatment with NPL-2008 targets repetitive behaviors that lock patients into set routines and hinders interest in normal social interaction.
Since it was formed in 2005, Autism Speaks has merged with two other U.S. autism charities. It set up the clinical trials network to speed up testing of drugs and other treatments for autism and said it can save considerable time in recruitment.
At the end of June, the charity announced $15.2 million in funding for 52 new research grants, spanning topics ranging from the risk factors for autism, to its underlying mechanisms, and treatment and prevention. Among the grants was funding for two placebo-controlled drug trials, one of melatonin, the second of the nicotinic antagonist mecamylamine, which has shown some promise in Tourette's syndrome and attention deficit hyperactivity disorder. Neuropharm, however, is funding the trial of NPL-2008 and will fully reimburse Autism Speaks for its services. It is the first industry-sponsored trial the charity has supported. Leatherhead, UK-based Neuropharm was founded in 2006 and joined the Alternative Investment Market in London in March, raising $38.4 million.