AMO Pharma Limited, a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, has entered a collaboration agreement with Ranedis Pharmaceuticals, a privately held U.S. biopharmaceutical company, to progress the development of RND-001 for rare genetic diseases, including certain lysosomal storage disorders and other diseases affecting the central nervous system. In addition, Ranedis has granted AMO an exclusive option to acquire the worldwide rights to RND-001 during the collaboration period.

RND-001 is a unique formulation of a histone deacetylase inhibitor (HDACi) whose properties allow for delivery of the molecule to the brain. In some genetic disorders, HDACis can result in increased or decreased transcriptional expression of mutated genes and confer other benefits associated with modulations of key transcriptional proteins. In pre-clinical research, RND-001 has been demonstrated to both stimulate gene transcription and protein expression in the brain and has shown potential efficacy in a mouse model of Neimann-Pick Type C disease (NPC). On this basis, AMO and Ranedis will research a range of applications in the treatment of rare diseases including certain neurodegenerative diseases for which there are currently no or limited treatments.

“RND-001 is a promising therapy supported by compelling early stage research that gives Ranedis Pharmaceuticals and AMO Pharma an excellent opportunity to leverage known mechanisms in previously inaccessible rare diseases,” said Michael Snape, chief executive officer, AMO Pharma.This collaboration represents an important next step in the expansion of our pipeline at AMO Pharma. RND-001 reflects many of the specific qualities we look for in asset acquisition, such as strong scientific rationale and opportunity to address unmet medical needs in rare diseases. We will continue to target new opportunities that fit our strategic focus.”

William Claypool, chief executive officer, Ranedis Pharmaceuticals, said, “The preclinical data have provided strong validation of the potential for RND-001 to uniquely address CNS symptoms in NPC and other rare diseases. We are pleased that the development program will advance under this agreement with AMO Pharma.”