Advisory board named to shepherd Duchenne MD candidate

Satellos Bioscience plans to start a first trial of SAT-3247 this year

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help propel SAT-3247, an oral therapy candidate for Duchenne muscular dystrophy (DMD).

“The formation of this clinical advisory board marks a major development step for Satellos as we continue our evolution in becoming a clinical stage drug development company,” Frank Gleeson, co-founder and CEO of Satellos, said in a company press release.

Satellos plans to initiate a first trial of SAT-3247 this year.

“We are proud and excited to bring together leading clinicians and scientists from across the world who are dedicated to, and have decades of experience in, the clinical development of novel therapeutics for degenerative muscle disorders. We believe this will help support Satellos in advancing our lead drug candidate, SAT-3247, as we work to optimize its potential to transform the treatment of Duchenne and serious muscle diseases,” Gleeson said.

Muscular dystrophy is made up of a group of genetic diseases that cause progressive muscle weakness and wasting.

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What is SAT-3247 and who is on the new advisory board?

SAT-3247 is a small oral molecule that inhibits the activity of AAK1, a protein that regulates the growth of muscle stem cells and is normally involved in muscle repair after damage. The therapy, which was developed using Satellos’ platform MyoReGenX, is meant to rescue the normal activity of muscle stem cells in people with muscular dystrophy, enabling muscle regeneration.

In a mouse model of facioscapulohumeral muscular dystrophy (FSHD), SAT-3247 significantly improved the function of skeletal muscles, which are used for movement. SAT-3247 was nominated as the company’s lead treatment candidate in DMD last year.

The new clinical advisory board’s seven members are:

  • Jordan Dubow, MD, chief medical advisor at Satellos with expertise in clinical and regulatory therapy development. Dubow has been pivotal in multiple applications for regulatory approval in the U.S., including Emflaza (deflazacort) for DMD.
  • Ronald Cohn, MD, PhD, president and CEO of The Hospital for Sick Children (SickKids) in Toronto. Cohn is a clinician and scientist working to develop new diagnostic tools and therapies, including the gene editing approach CRISPR.
  • Richard Finkel, MD, director of the Center for Experimental Neurotherapeutics at St. Jude Children’s Research Hospita in Memphis. Finkel has nearly three decades of experience pursuing treatments for neurologic disorders, leading clinical trials for neuromuscular diseases, including DMD and spinal muscular atrophy.
  • Nicholas Johnson, MD, director of the Center for Inherited Myology Research, and vice chair of research in neurology at Virginia Commonwealth University. His lab is working to identify the mechanisms that underlie muscular dystrophies. Johnson has been involved in clinical trials in inherited nerve and muscle disorders.
  • Hanns Lochmüller, MD, PhD, a neurologist and a senior scientist at the Children’s Hospital of Eastern Ontario Research Institute, Canada. His research interests span the mechanisms of and therapy development for neuromuscular disorders and his clinical practice focuses on rare neuromuscular disorders, including myotonic dystrophy and spinal muscular atrophy.
  • Francesco Muntoni, MD, director of the Dubowitz Neuromuscular Centre at the Great Ormond Street UCL Institute of Child Health, London, his work is focused on pediatric neuromuscular disorders, especially DMD. He played a part in the studies that supported the approval of Exondys 51 (eteplirsen) and Vyondys 53 (golodirsen) for DMD, and in identifyiing more than 30 neuromuscular disease genes.
  • Perry Shieh, MD, PhD, a neurologist and a professor of neurology at UCLA David Geffen School of Medicine. Shieh has served as researcher in numerous clinical trials for neuromuscular conditions. His clinical interests include DMD, FSHD, and myotonic dystrophy.