Building a Portfolio of High Impact New Medicines

AMO Pharma’s two investigational drugs currently in development, AMO-01 and AMO-02, are both positioned to advance to phase 2 clinical trials, or proof-of-clinical-concept studies in the relevant patient populations. AMO-01 is in development for treatment of fragile X syndrome and AMO-02 is in development for treatment of congenital myotonic dystrophy. Prior to being acquired by AMO Pharma, the safety profiles of both AMO-01 and AMO-02 were extensively characterized in previous pre-clinical studies for other indications. Their safety profiles and demonstrated mechanisms of action strongly support their development in these indications and also show that they have potential for use in treatment of certain other orphan and non-orphan diseases.