SAT-3247 improves muscle function in FSHD mouse model

Therapy is also lead candidate for Duchenne MD

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by Andrea Lobo |

A trio of mice are shown rummaging around bottles of medicine.

SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD).

Developed by Satellos Bioscience, the therapy is the lead treatment candidate for Duchenne muscular dystrophy (DMD), with the first clinical trial of SAT-3247 for DMD expected to start this year.

“We look forward to presenting these data at the MDA Clinical and Scientific Conference 2024 and advancing SAT-3247 into clinical trials for Duchenne muscular dystrophy mid-year,” Frank Gleeson, Satellos’ co-founder and CEO, said in a press release.

Muscular dystrophy comprises a group of genetic diseases that cause progressive muscle weakness and wasting. In FSHD, the muscles of the face, shoulders, and upper arms are typically affected. The disease occurs when a region of DNA called D4Z4, which includes the DUX4 gene, is activated, leading to the DUX4 protein being produced in cells, where it is not normally produced. This is thought to be toxic, particularly to muscle cells.

SAT-3247 is a small molecule that’s designed to inhibit the activity of a protein called AAK1, which regulates the growth of muscle stem cells and is normally involved in muscle repair after damage.

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The therapy is expected to normalize the activity of muscle stem cells in people with muscular dystrophy, enabling muscle regeneration. It was developed through Satello’s discovery platform MyoReGenX, which powers the discovery of new candidates for treating degenerative muscle conditions.

In a research project funded by the FSHD Canada Foundation, Satellos tested whether SAT-3247 could work against muscle destruction in a mouse model of FSHD. The results demonstrated that treatment with SAT-3247 significantly improved the skeletal muscle function of FSHD mice.

“These data in FSHD, in combination with our preclinical Duchenne studies, collectively demonstrate the impact of SAT-3247 on improving muscle regeneration in degenerative muscle diseases,” Gleeson said.

“It’s early days, but these results provide hope for FSHD patients. I’m looking forward to working with Satellos to accelerate the development of this promising drug candidate, because time is muscle!” said Neil Camarta, FSHD Canada Foundation’s co-founder and CEO.

Another AAK1-targeting compound, named SAT-3153, is another potential treatment for DMD from Satellos. It was granted orphan drug status and rare pediatric disease status by the U.S. Food and Drug Administration. SAT-3153 is intended to serve as backup to SAT-3247.