AMO Pharma has reported positive results in a Phase II trial of AMO-02 for the treatment of type 1 congenital and childhood onset myotonic dystrophy (CDM1).
Results of the proof-of-concept trial have demonstrated AMO-02 to be safe and well tolerated, with no early discontinuations or dose adjustments required.
In the trial, most patients treated with AMO-02 also showed clinical benefits such as improvements in cognitive function, levels of fatigue and the ability to perform daily tasks.
The trial further showed greater response in patients who received 1000mg/day of AMO-02 versus the 400mg/day of AMO-02 dose.
The results also showed co-occurring autism symptoms improved in various AMO-02 patients.
AMO Pharma CEO Michael Snape said: “These significant data are an important step in the development of AMO-02 as a potentially safe and effective treatment option for many patients living with congenital and childhood onset myotonic dystrophy type 1.”
The trial was primarily designed to evaluate the safety and tolerability of AMO-02 in 16 patients aged 13 to 34 years old, who suffer from type 1 myotonic dystrophy.
Over a 14-week treatment period, patients received either an oral dose of placebo, 1000mg/day of AMO-02 or 400mg/day of AMO-02.
AMO-02 is an investigational medicine which is yet to be approved for use in the treatment of myotonic dystrophy, a genetic disease that typically presents itself at birth.
People living with CDM1 generally suffer from weakened muscles and difficulties with thinking, problem solving, speech, hearing and vision.
No approved therapies are currently available for CDM1.