deCODE chemistry and Families of Spinal Muscular Atrophy (FSMA) today announced a renewal of their collaboration to develop a small molecule therapeutic for spinal muscular atrophy, a genetic disorder that is the leading killer of children under two years of age. Initiated in 2003, the deCODE chemistry-FSMA collaboration focused on optimization of a class of molecule that was discovered in a high-throughput, cell-based phenotypic assay developed under sponsorship by FSMA. By providing services for lead optimization and in-house support of the phenotypic assay, deCODE chemistry has developed optimized analogues that have high potency in the assay, excellent metabolic stability, efficient penetration of the blood-brain barrier, and an attractive pharmacokinetic profile. The focus of the collaboration in the coming year will be to further assess the pharmaceutical properties of lead candidates and select a small number of analogues for IND-directed, pre-clinical development.

“We are pleased to continue our collaboration with FSMA on this important program” said Dr. David Zembower, Vice President of Chemistry at deCODE chemistry. “We are encouraged by the progress that has been made over the past year, and look forward to working with FSMA to help them achieve their goals.”

“There has been much progress in SMA research since our relationship with deCODE began, and we are proud that our collaborative effort has been a part of that,” said Dr. Jill Jarecki, FSMA Research Director. “We are confident that our pioneering endeavor has the potential to enable a novel treatment for SMA.”