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AMO Pharma Announces Initiation of Clinical Trial conducted by Mount Sinai to Study AMO-01 in Treatment of Phelan-McDermid Syndrome

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Tuesday, May 08, 2018

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LONDON and PHILADELPHIA - AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, debilitating childhood onset neurogenetic disorders with limited or no treatment options, announced initiation of patient recruitment in an interventional study of AMO-01, the company's investigational Ras-ERK pathway inhibitor, in the treatment of Phelan-McDermid syndrome. The clinical trial is being conducted by the Icahn School of Medicine at Mount Sinai (ISMMS) in New York.

"Treatment of Phelan-McDermid syndrome represents a significant area of unmet need in healthcare, and AMO Pharma is grateful to the research team at Mt. Sinai as well as the Phelan McDermid Syndrome Foundation for their commitment to this landmark research effort," said Michael Snape, chief executive officer of AMO Pharma. "Research thus far indicates that AMO-01 could have important applications in the treatment of patients living with Phelan-McDermid syndrome in the years ahead."

AMO Pharma will provide AMO-01 and grant funding for this investigator led study in subjects with Phelan-McDermid syndrome aged 12 years or older who have a history of epilepsy. Phelan-McDermid syndrome is a rare genetic condition caused by a chromosomal deletion. The most common characteristics found in these patients are intellectual disability of varying degrees, delayed or absent speech, symptoms of autism spectrum disorder, low muscle tone, motor delays and epilepsy.

"We are very pleased to be leading this important clinical research effort in collaboration with AMO Pharma," said principal investigator Alexander Kolevzon, M.D. "In addition to potentially leading to a new treatment option for patients, this research effort can also provide us with many important new insights about the onset, progression and management of Phelan-McDermid syndrome."

About AMO-01
Recent studies suggest that extracellular signal-regulated kinase (ERK) plays a critical role in synaptic plasticity (the ability of certain synapses to strengthen or weaken over time) and neurodevelopment. AMO-01 is an inhibitor of the Ras-ERK pathway. In pre-clinical efficacy studies, AMO-01 has been shown to rescue the neuronal phenotype of the multiple knockout mouse models of intellectual disability. This provides strong scientific evidence to support the conclusion that inhibition of the Ras-ERK cascade may have therapeutic benefit in the treatment of intellectual disabilities in humans.

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

About Phelan-McDermid Syndrome
Phelan-McDermid syndrome (PMS) is also known as 22q13 Deletion Syndrome. It is a genetic condition that is caused by a mutation of the SHANK3 gene or a deletion (a missing piece) of genetic material that causes many different but related symptoms. The genetic changes that cause PMS vary from person to person and can occur randomly or be inherited from a parent who carries a related genetic change. Because the genetic changes vary, the symptoms of PMS vary too, and can cause a wide range of medical, intellectual, and behavioral challenges. The most common characteristics found in those with PMS are intellectual disability of varying degrees, delayed or absent speech, symptoms of autism spectrum disorder, low muscle tone, motor delays, and epilepsy. There is currently no cure or treatment specifically for PMS. Current treatments help manage many of the symptoms and researchers are working hard to improve our knowledge of PMS and to find drugs and therapies that can help people affected by PMS.

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

Kelly Wakelee
Berry & Company Public Relations

Source: AMO Pharma Ltd.
1.3 from 3 votes
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