Satellos nominates SAT-3247 as lead treatment candidate for DMD

Developer hopes to get FDA OK to launch clinical trials next year

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Satellos Bioscience is hoping to bring SAT-3247, its newly nominated lead treatment candidate for Duchenne muscular dystrophy (DMD), into clinical trials next year.

The company announced that it’s working to establish a good manufacturing practice (GMP) setup to make the medication, and is in the midst of experiments to support an investigational new drug application (IND) to the U.S. Food and Drug Administration (FDA). An IND is a formal request to the U.S. regulatory authority for approval to start testing a treatment candidate in people.

“We look forward to expeditiously completing our ongoing IND-enabling studies and GMP manufacturing and moving into clinical trials,” Phil Lambert, PhD, chief scientific officer of Satellos, said in a company press release.

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SAT-3247 is designed to block the activity of a protein called AAK1, which helps to regulate the growth of muscle stem cells. Normally, these stem cells are able to grow and create new muscle fibers to help repair muscle damage in the body. But in DMD, their ability to repair muscle damage is impaired.

By blocking AAK1, SAT-3247 is expected to help normalize the activity of muscle stem cells to promote the repair of muscle tissue in DMD, according to Satellos.

“We identified AAK1 as having the potential to affect muscle regeneration by regulating asymmetric muscle stem cell polarity, independent of dystrophin [the protein whose defect causes DMD],” said Frank Gleeson, co-founder and CEO of Satellos.

According to Lambert, the company’s “data … continue to support our drug target, mechanism of action, and proprietary small molecule leads.”

“In addition, previously published external clinical programs with AAK1 inhibitors have demonstrated the ability to safely inhibit this target, which further encourages us in our development plans,” Lambert said.

Satellos had previously announced another AAK1-targeting experimental compound, dubbed SAT-3153, as a potential muscle regeneration treatment for DMD. Earlier this year, that therapy was designated an orphan drug by the FDA, a status that aims to expedite development.

However, according to the company, while both therapies showed a similar ability to modulate muscle stem cell activity and improve muscle force in mice, SAT-3247 was better at getting to muscle tissue after being taken by mouth.

That led to SAT-3247’s selection as the company’s lead treatment candidate for DMD. SAT-3153 will serve as a backup candidate.

“With our team’s expertise, we have created small molecules that are selective for and effective at inhibiting AAK1 to modulate muscle stem cell polarity leading to the repair and regeneration of functional muscle tissue in our preclinical models. We are excited about the improved oral bioavailability, target selectivity, and tissue distribution that SAT-3247 has exhibited in these models,” Lambert said.

Satellos also announced that Courtney Wells, a 20-year industry veteran who has experience with clinical trials in DMD, has been tapped as its new senior vice president of clinical development operations.

“We are also pleased to welcome a clinical development professional of Courtney’s stature and experience who chose to join Satellos and lead the execution of our clinical development plan for SAT-3247 at this critical junction in our evolution to becoming a clinical stage company in 2024,” Gleeson said.

Alan Jacobs, MD, chief medical officer of Satellos, added that Wells’ leadership, “will be invaluable in leading and advancing SAT-3247 through clinical development, which we plan to initiate in mid-2024.”