Dr. Elliott Androphy, a member of Spotlight’s scientific advisory board, will lead the joint effort. Androphy is a co-inventor of STL-182, Spotlight’s oral small molecule, which has been shown to benefit SMA patients.
SMA is an autosomal recessive disorder, which means that it only develops if a child inherits no intact spinal motor neuron 1 (SMN1) gene from either parent. In children with SMA, low levels of a functional SMA protein can be produced by an SMN1-related gene, called SMN2. STL-182 stimulates production of the SMN protein encoded by SMN2.
Spotlight, based in Urbandale, Iowa, has previously reported that STL-182 can restore neuromuscular function in SMA mouse models by stabilizing such SMN protein levels. Androphy and Kevin Hodgetts, director of the Laboratory for Drug Discovery in Neurodegeneration at Boston’s Brigham and Women’s Hospital, collaborated in preclinical studies related to those findings.
Androphy, chair of the dermatology department at Indiana University’s School of Medicine in Indianapolis, has used a novel, cell-based high throughput strategy to screen for compounds that increase SMN protein levels – work that has led to the identification of pre-clinical drug candidates for SMA.
He also characterized the disease-causing mechanism of alternative splicing of the SMN2 gene as a vice-chair for research at the University of Massachusetts Medical School.
“Dr. Androphy is a prolific researcher and highly respected thought leader,” Geoffrey Laff, Spotlight’s senior vice president of business development, said in a press release. “We are privileged to work with him to develop novel therapies for SMA.”
Androphy has published research in Nature, Human Molecular Genetics, Molecular Cell and other high-profile scientific journals.