Sepul Bio, an innovative business unit of Laboratories Théa dedicated to advancing therapies for inherited retinal diseases, today announced the enrollment of the final participant in the Phase 2b LUNA clinical trial of Ultevursen in patients with Usher syndrome type 2A (USH2A)-associated retinitis pigmentosa (RP) or non-syndromic RP.
“Achieving the enrollment of the final participant in the Phase 2b LUNA clinical trial represents a fantastic milestone for USH2A research,” said Dr. Robert Koenekoop, MD, PhD, Professor in the Department of Pediatric Surgery at the Faculty of Medicine and Health Sciences, McGill University.
“This moment takes us one step closer in our search for therapies for this condition of high unmet need. The LUNA clinical trial, being conducted across 26 active clinical sites in 10 countries, is exploring the impact of RNA therapies on individuals with USH2A-associated RP due to variants in exon 13 of the USH2A gene.”
“We are thrilled to see the full enrollment of the LUNA clinical trial,” said Krista Vasi, MPA, Executive Director of the Usher Syndrome Coalition.
“The Usher Syndrome Coalition has supported the LUNA trial from protocol review through recruitment, connecting eligible families through the USH Trust, the largest international contact database of individuals with Usher syndrome, and amplifying this trial for families living with a USH2A exon 13 variant.
“This milestone brings our partnership full circle as we now enter the waiting period for data at the end of this trial. Each enrolled trial is a meaningful step forward toward treatments for our community and, one day, therapies that can change the lives of children, adults and families living with Usher syndrome type 2A.”
“There are currently no treatments for inherited retinal diseases such as Usher syndrome,” said Professor Katarina Stingl, Head of the Clinic for Hereditary Retinal Degeneration at the Center for Rare Eye Diseases, University Hospital Tübingen.
“I am delighted the LUNA clinical trial has now fully enrolled, as it marks a moment in Ultevursen’s clinical development when we can look forward to emerging data and real results for the USH2A community. I look forward to working with the Sepul Bio team to hopefully bring forward a treatment that can slow disease progression or even reverse it in families affected by USH2A-associated RP.”
Retinitis pigmentosa is caused by one or more variants, or defects, in a gene in the DNA, which is then copied to RNA. This mutation disrupts the production of an important protein in the retina called usherin, which is believed to cause photoreceptor death and consequent vision loss.
Ultevursen, an investigational RNA therapy, will be evaluated for safety and efficacy in the clinical trial. It is designed to overcome this genetic defect and restore the important protein, potentially slowing or stopping some of the effects of RP.
Ultevursen is specifically designed to target mutations in exon 13 of the USH2A gene. Hybridization of Ultevursen to the pre-messenger ribonucleic acid, or pre-mRNA, modulates the RNA splicing process, leading to the exclusion of exon 13 from the mRNA.
The exclusion of exon 13 results in the production of a functional, shorter usherin protein. It is hypothesized that producing the usherin protein in the retina could potentially prevent or slow the progression of photoreceptor degradation and thereby preserve visual function in patients.
LUNA, also known as SB-421a-006, is a two-year, double-masked, randomized, sham-controlled trial of Ultevursen for adults and children with retinitis pigmentosa caused by mutations in exon 13 of the USH2A gene.
The study includes children over eight years of age globally and those aged 12 years and older in Europe. Available safety and efficacy data from previous Ultevursen clinical studies support its therapeutic potential.
As an innovative business unit of Théa, Sepul Bio is at the forefront of advancing transformative RNA therapies for inherited retinal diseases, with particular emphasis on the further development of two cutting-edge ophthalmic products, Sepofarsen and Ultevursen.
Sepofarsen targets Leber congenital amaurosis 10 (LCA10), a rare genetic condition that causes severe visual impairment, while Ultevursen is designed to halt vision loss in individuals with a mutation in exon 13 of the USH2A gene who are living with retinitis pigmentosa.
Sepul Bio’s programmes are driven by a vision of a future in which patients with inherited eye diseases have treatment options for their conditions. Through ongoing research and rigorous development, Sepul Bio hopes to bring new therapies to patients.
Théa is the leading independent European pharmaceutical company specializing in the research, development and commercialization of eye care products.
Based in Clermont-Ferrand, France, this family-owned and operated company comprises approximately 2,400 collaborators. Théa has expanded by opening more than 35 affiliates and offices across Europe, North Africa, North and South America, and the Middle East. Its products are available in 75 countries.