After receiving a favorable opinion from the ANSM for its clinical study “AVANCE 1”, SQY Therapeutics announces today the start of the recruitment of patients who will participate in the first administrations in humans (First-in-Human) of the compound SQY51, a next-generation antisense oligonucleotide, designed to restore a semi-functional dystrophin by skipping exon 51 of the DMD gene in a sub-population of boys affected by Duchenne Muscular Dystrophy.
SQY Therapeutics, the sponsor of the clinical trial “Avance1”, is pleased to announce that its clinical trial authorization application has been accepted by the ANSM (Agence Nationale de Sécurité des Médicaments et des Produits de Santé) under the European regulation on clinical trials of medicinal products, number EU CT 2022-500703-49-01, and that patient recruitment has started.
“Avance1” is a single-center phase 1/2a study aimed at evaluating the safety, pharmacokinetics, and pharmacodynamics of SQY51 in pediatric and adult patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy. Twelve patients (aged over 6 years) will be included in the trial. During the 13-week phase 1 of the study, they will all receive 6 ascending doses of SQY51 intravenously. For phase 2a, the phase 1 participants will be divided into 3 cohorts, each treated with a different dose of SQY51 for 32 weeks.
A set of preclinical studies indicates that SQY51 has a satisfactory safety profile and that it is capable of reaching all the organs and tissues affected by the disease. As the chemistry of tricyclo-DNA has not yet been evaluated in humans, the “Avance1” trial is qualified as “First-in-Human” and the participants exposed to the drug candidate will be monitored with the utmost attention. The expected result is that SQY51 will restore dystrophin production where it is needed, in order to slow down or even stop the progression of the disease in Duchenne patients concerned by the skipping of exon 51.
The drug candidate SQY51 is the result of a fruitful collaboration between SQY Therapeutics and the research unit UMR1179 Inserm-UVSQ led by Luis Garcia at the UFR Simone Veil – Santé of the University of Versailles Saint-Quentin-en-Yvelines (Université Paris-Saclay).
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disease causing progressive degeneration of all the muscles in the body. It is caused by abnormalities in the DMD gene (mutation), located on the X chromosome, and coding for dystrophin, a protein essential for the proper functioning of muscle fibers. The disease affects about one in 3500 boys at birth. It results in inexorable and highly disabling muscle degeneration. Generally, boys lose the ability to walk between the ages of 10 and 13, and respiratory assistance is often necessary from adolescence. The involvement of the cardiac muscle is life-threatening.
About SQY51
SQY51 is an antisense oligonucleotide designed to hybridize to a specific site on the pre-mRNA transcribed from the DMD gene, so that certain elements of the mutated gene are excluded during mRNA maturation (exon skipping) in order to restore the production of a functional truncated dystrophin. Most often, antisense oligonucleotides are small chains of 15 to 30 nucleotides (bases A, C, G, T) chemically modified to ensure their stability in the body. SQY51 uses non-natural nucleotides from the “tricyclo-DNA” family. They have the advantage of being very stable and hybridizing more effectively with their RNA target than their natural counterparts.
About SQY Therapeutics
SQY Therapeutics is a young biotechnology company initiated by parents of boys with myopathy and researchers whose goal was to implement R&D programs with a clinical focus for genetic diseases, particularly for Duchenne Muscular Dystrophy. The start-up SQY Therapeutics was created in 2015 and hosted at the UFR Simone Veil – Santé of the UVSQ until it moved into its own premises. Established close to the scientific and hospital-university environment of the UVSQ, SQY Therapeutics develops, in the territory of Saint-Quentin-en-Yvelines, an unparalleled technological platform focused on tricyclo-DNA with the perspective of promoting new therapeutic solutions for highly disabling diseases for which the care offer remains unsatisfactory to this day.
