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AMO Pharma Shows Weakness In Myotonic Dystrophy

 
• By Elizabeth Cairns

A pivotal miss leaves the company more reliant on earlier-stage pipeline programs as it talks to regulators about the path forward for its lead product.

A man shows a thumb down. Gesture of disapproval and rejection. Bad assessment, harsh criticism, failure and defeat. Refusal to pass and cooperation. Bureaucratic obstacles. Contempt and condemnation

The private UK group Amo Pharma Limited is hoping to bring to market the first disease-modifying therapy for congenital myotonic dystrophy type 1 (DM1). This will be a difficult task: on Saturday the company disclosed that the product’s pivotal trial had failed. 

DMI is characterized by muscle weakness and wasting, prolonged muscle tensing, cataracts and abnormal heart function, and can be fatal. It is caused by extra copies of a repeating sequence of three nucleotides in the noncoding region of the DMPK gene

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