Spotlight Innovation has obtained an exclusive, worldwide license to STL-182, a novel compound invented by Drs. Elliot Androphy at Indiana University and Kevin Hodgetts, director of the Laboratory for Drug Discovery in Neurodegeneration at Brigham and Women’s Hospital. The investigators’ successful research collaboration was supported in part by a series of grants from the National Institutes of Health.

Spotlight Innovation plans to develop and commercialize STL-182 for treatment of patients with spinal muscular atrophy (SMA). STL-182 is an orally-available small molecule that stabilizes SMN protein, causing it to last longer.

Dr. Androphy is a member of the Cure SMA Scientific Advisory Board and, together with Dr. Hodgetts, recently received a grant from Cure SMA for development of another novel series of compounds. These compounds target SMN2, the SMA “backup gene,” in an effort to prompt SMN2 to produce more SMN protein. “I truly appreciate all the support of Cure SMA has provided over the years and am hopeful that this work will come to fruition for those affected by SMA,” said Dr. Androphy.

Developing Combination Therapies to Attack SMA from All Sides

The SMN2 gene only produces a small amount of fully functional SMN protein due to a splicing error. Many of the compounds already in development target this splicing error, with the goal of increasing the amount of functional SMN protein in the body. The two compounds that Drs. Androphy and Hodgetts are developing address this same issue through other means: one to prompt SMN2 to make more protein and one (the compound licensed by Spotlight) to make that protein last longer. Researchers believe all three approaches may be used in combination with each other. This would open up yet another possible avenue for combination therapies, which are needed to develop treatments for all ages, types and stages of SMA.

Congratulations to Drs. Adrophy and Hodgetts on their fine work!

The Importance of Leverage

It can cost upwards of $1 billion to bring a therapy through the development process to FDA approval. This level of investment requires collaboration between academics, industry, government, and families. Cure SMA brings all of those groups together, breaking down roadblocks that might delay important developments.

One of our most powerful tools in breaking down those roadblocks is our “seed funding” approach. We provide early-stage funding for promising new ideas and novel treatment approaches. In doing so, we lower the risk and attract larger investments from industry, government, and other funding sources as drug candidates move through the process.

In addition, we are working to increase the number of new therapies in the SMA pipeline, to insure we have treatments for all ages and stages of SMA. Attracting investment from government, pharmaceutical companies, and other organizations provides the resources necessary to accomplish these goals.

Pictured: Dr. Androphy participating in the Researcher Relay Race at the Annual SMA Conference.