Building a Portfolio of High Impact New Medicines

AMO Pharma's investigational drugs currently in development include:


  1. AMO-01 for the treatment of intellectual disabilities.
  2. AMO-02 for the treatment of congenital myotonic dystrophy.
  3. AMO-04 for the treatment of Rett syndrome.
  4. RND-0001 for the treatment of certain lysosomal storage disorders.

AMO-01, AMO-04 and RND-0001 are positioned to advance to phase 2 clinical trials or proof-of-clinical-concept studies in the relevant patient populations. AMO-02 is near the end of a first phase 2 clinical trial. Prior to being acquired by AMO Pharma, the safety profiles of all of the assets in our pipeline were extensively characterized in previous 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.