AMO Pharma Receives FDA Orphan Drug Designation for AMO-04 in Treatment of Rett Syndrome

LONDON and PHILADELPHIA, June 13, 2018 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, debilitating diseases with limited or no treatment options, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its investigational therapy AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.

"This designation is an important milestone in our development program for AMO-04 and reinforces the critical need for a new treatment option for Rett syndrome, which causes devastating neurological regression in patients," said Michael Snape, chief executive officer of AMO Pharma. "We look forward to advancing this program into the clinic and furthering our commitment to this underserved patient population in which there are currently no approved therapies." 

Rett syndrome is a neurological disorder that is typically first recognized in infancy and seen almost always in girls. It is caused in most cases by mutations in the X-linked methyl-CpG-binding protein 2 (MECP2) gene. It is often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay because patients typically experience problems with cognitive, sensory, emotional, motor and autonomic function. Other symptoms include seizures, disorganized breathing patterns while awake, scoliosis and sleep disturbances.

AMO-04 is a glutamate modulator that has shown early-stage promise in the treatment of Rett syndrome based on screening by the Scout Program, a drug discovery screen in a mouse model of the disease that is sponsored by Additional research conducted by Numedicus, a private biopharmaceutical company focused on the repurposing of drugs, also indicated AMO-04 could benefit patients living with certain breathing disorders. AMO Pharma has an exclusive license agreement with Numedicus covering AMO-04 and related compounds.

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, debilitating childhood onset neurogenetic disorders with limited or no treatment options. For more information, please visit the AMO Pharma website at

Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448

Kelly Wakelee
Berry & Company Public Relations

SOURCE AMO Pharma Limited

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