LONDON and PHILADELPHIA, June 7, 2016 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today presented findings from several pre-clinical research studies demonstrating that AMO-01, one of the company's investigative therapies, successfully inhibits activation of the Ras-ERK intracellular pathway, a pathway that is integral to the pathophysiology associated with progression of fragile X syndrome. Results were presented in a poster session during the Gordon Research Conference Fragile X and Autism-Related Disorders Meeting.
The presentation, titled 'The Effect of AMO-01 on the Fmr1 Knockout Mouse Model of Fragile X Syndrome', highlighted results from multiple pre-clinical efficacy studies. Data results showed AMO-01 successfully reversed abnormalities in neuronal anatomy and cognitive and behavioral deficits in transgenic mouse models of fragile X syndrome. This broad profile of efficacy was seen after a single treatment with AMO-01 and persisted afterward. AMO Pharma is planning for AMO-01 to be the first drug candidate with this mechanism to enter clinical trials.
"The Ras-ERK pathway plays a central role in the progression of fragile X syndrome, and the results from multiple pre-clinical studies show AMO-01 successfully inhibits this activated pathway. Based on these findings, we see strong support for our plan to advance AMO-01 to clinical development in 2016 for the treatment of fragile X syndrome," said Michael Snape, chief executive officer of AMO Pharma.
"FRAXA is encouraged by AMO's development of AMO-01 for the treatment of fragile X syndrome. These data results provide insight into the role of the Ras-ERK pathway in fragile X syndrome, support the advancement of AMO-01 into clinical studies and offer hope as we strive to develop new treatment options for patients as quickly as possible," said Dr. Michael Tranfaglia, medical director and chief scientific officer of FRAXA. FRAXA is an international nonprofit organization focused on finding a cure for fragile X syndrome.
About Fragile X Syndrome
Fragile X syndrome is the most common inherited cause of autism and intellectual disabilities, affecting approximately 1 in 4,000 males and 1 in 6,000 females. Symptoms range in severity and can include intellectual disabilities, attention deficit and hyperactivity, anxiety and unstable mood, sensory integration problems, such as hypersensitivity to loud noises or bright lights, speech delay and seizures. There are currently no approved therapies indicated for the treatment of fragile X syndrome.
About AMO Pharma
AMO Pharma is a biopharmaceutical company incorporated in February of 2015. The co-founder, Dr. Michael Snape, has extensive experience in senior scientific and operational roles in both large pharma and biotech companies spanning more than twenty five years, and has brought together a targeted and experienced senior management team with a proven track record of success in all phases of product development and acquisition. The company is working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare genetic diseases. AMO Pharma is currently advancing two investigational therapies for treatment of fragile X syndrome and myotonic dystrophy, and is in the process of licensing additional development-stage products in diverse areas including autism and other CNS disorders. For more information, please visit the AMO Pharma website at http://www.amo-pharma.com/.
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SOURCE AMO Pharma Limited