AMO Pharma Announces FDA Fast Track Designation For AMO-02 For Treatment Of Congenital Myotonic Dystrophy

Tuesday, May 30, 2017 Drug News
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LONDON and PHILADELPHIA, May 30, 2017 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"),

a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AMO-02, the company's investigational
therapy in development for the treatment of congenital myotonic dystrophy. 

Fast Track designation is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Drugs that receive Fast Track designation are eligible for more frequent meetings and written communications with the FDA, accelerated review and priority approval, and rolling New Drug Application review.

"Congenital myotonic dystrophy is a devastating neuromuscular disease where affected patients currently have no treatment options available," said Michael Snape, chief executive officer of AMO Pharma. "The designation of Fast Track status for our development program for AMO-02 highlights both the urgent need for a treatment for patients and the potential for this novel therapy to offer a major advance in their care in the years ahead."

AMO-02 (tideglusib) is in clinical stage development for the treatment of the severe form of myotonic dystrophy known as congenital DM1. In cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to be increased. Inhibitors of GSK3ß have been shown to correct the activity of RNA binding proteins, such as CUGBP1, in animal models of DM1. AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples derived from patients with congenital DM1.

"Our ongoing phase 2 clinical trial for AMO-02 in the UK is the first sponsor-led clinical study in the treatment of congenital myotonic dystrophy and represents a historic milestone in research and a new era of hope for patients and their families affected by this serious condition," Snape added.  "In addition to advancing this development program, our research will provide many new insights to help shape the future of new research and to offer the prospect of better health for people living with myotonic dystrophy."

About Myotonic Dystrophy Myotonic dystrophy is the most common form of muscular dystrophy, affecting approximately 1 in 10,000 people. It is a progressive disease and the predominance of symptoms such as muscle weakness, intellectual/developmental impairment or cognitive/behavioral changes is determined by a number of factors, including age at onset. There are currently no approved therapies indicated for the treatment of myotonic dystrophy.

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


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

Media: Sara Zelkovic Berry & Company Public Relations 212 253 8881


To view the original version on PR Newswire, visit:

SOURCE AMO Pharma Limited

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