AMO Pharma Sees Positive Results in Phase II Trials

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AMO Pharma reported that patients treated with its Phase II neuromuscular treatment for onset myotonic dystrophy type 1 experienced clinical benefits such as an improvement in cognitive function and an increased ability to perform daily tasks.

The privately-held biopharmaceutical company shared final data from its mid-stage proof-of-concept study this week during the Muscular Dystrophy Association Conference in Virginia. AMO said AMO-02 was safe and well tolerated. There were no early discontinuations or dose adjustments required during the trial, the company said.

AMO-02 (tideglusib) is an inhibitor of glycogen synthase kinase 3 beta (GSK3ß). In patients battling neuromuscular diseases, such as congenital myotonic dystrophy and even Duchenne muscular dystrophy, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. During preclinical testing, AMO said its GSK3ß inhibitor was shown to correct the activity of regulatory proteins, such as CUGBP1.

“Inhibition of increased levels of GSK3ß is known to reverse cognitive and behavioral deficits in transgenic models of syndromic autism, a developmental disorder characterized by social communication deficits and repetitive behaviors,” AMO said on its website.

During its presentation at the conference, AMO said there was a greater response in patients who received a dose of 100 mg versus a higher dose of 400 mg. Results also showed co-occurring autism symptoms improved in several AMO-02 patients, AMO announced.

Myotonic dystrophy type 1 is a rare, genetic, life-threatening neuromuscular disorder that causes impairment in muscle function, cognition and quality of life. Myotonic dystrophy is the most common form of muscular dystrophy, affecting approximately 1 in 8,000 people. There are currently no approved therapies indicated for the treatment of myotonic dystrophy. 

AMO Chief Executive Officer Michael Snape said the significant data the company reported from its Phase II trial shows the medication is a potentially “safe and effective treatment option” for patients with congenital and childhood-onset myotonic dystrophy type 1.

“We look forward to advancing the clinical development program for AMO-02 and are grateful to the clinicians, caregivers and patients who participated in this landmark trial,” Snape said in a statement.

Joseph Horrigan, AMO’s chief medical officer, said the Phase II trial was the first devoted to that particular patient population. He said the results are encouraging and the company will continue to expand its research to include larger multi-site clinical trials in the United States, Canada and the United Kingdom.

In July 2017 the U.S. Food and Drug Administration granted Orphan Drug Designation for AMO-02. The designation is granted to developmental treatments for rare diseases/disorders that affect fewer than 200,000 people in the U.S.

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