Clinical Trial Assessing Novel Antisense Oligonucleotide for DMD Underway

A new clinical trial, AVANCE1, is recruiting adult and pediatric patients with Duchenne muscular dystrophy (DMD) to investigate the safety, pharmacokinetics (PK), and pharmacodynamics of a novel antisense oligonucleotide called SQY51.

The trial, sponsored by SQY Therapeutics and Biotrial, is recruiting up to 12 participants to initially receive escalating intravenous infusions of SQY51 every 2 weeks during the 13-week phase 1 portion of the trial. Patients will then be enrolled in the 32-week phase 2a of the trial, where they will be allocated to 1 of 3 cohorts in a nonrandomized manner to receive 1 of 3 treatment doses in 4 blocks of 4 weeks.

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The study will measure the incidence of adverse events in all participants as its primary outcome. Secondary outcome measures will include PK plasma concentrations of SQY51, changes in functional measures, and changes from baseline in skeletal muscle dystrophin expression.

Some of the functional measures observed will be changes from baseline in time to rise from the floor and distances completed during 1-minute, 6-minute, and 10-minute walk tests in ambulatory patients. Changes in motor function measure scores and performance of upper limb scores will also be compared with baseline values for both ambulatory and nonambulatory patients.

The study is recruiting patients at a single clinical site in Garches, France. Participants must be at least 6 years of age and have a genetic diagnosis of DMD amenable to exon 51 skipping.

“SQY51 is a very promising molecule for the treatment of boys with Duchenne muscular dystrophy who are eligible for exon 51 skipping. I have been involved in the development of this drug for 15 years, which is a result of a close collaboration between parents of boys suffering from DMD, researchers, and doctors,” Helge Amthor, MD, PhD, principal investigator of the study, said.