SparingVision has reported positive initial safety results from the first cohort treated with gene-agnostic gene therapy SPVN06 in the Phase I/II PRODYGY trial for retinitis pigmentosa (RP).
The multicentre study is designed to assess the tolerability, safety, preliminary efficacy and quality-of-life in adult patients with rod-cone dystrophy (RCD) due to a mutation in the RHO, PDE6A, or PDE6B gene.
It includes an open-label, dose-escalation and controlled, double-masked, randomised extension phases.
The study’s principal investigators are Isabelle Audo at the 15-20 National Hospital in Paris, France, and Dr Joseph Martel of the Eye Center from the University of Pittsburgh Medical Center in Pittsburgh, US.
Out of the total 33 adults, three patients with severe advanced RCD in the first cohort received a single subretinal injection of low-dose SPVN06 at the 15-20 National Hospital.
SPVN06 showed a favourable safety profile and was well tolerated in these patients.
The Data Safety Monitoring Board reviewed the safety data and recommended that it is safe to dose the second cohort with a medium dose.
For the second cohort, the company intends to enrol 24 patients with intermediate advanced RP with six untreated subjects while 18 patients will receive one of the two recommended doses from the first cohort study.
The primary endpoint of the study is anticipated to be achieved in 2025 while preliminary efficacy data is expected in the same year.
SparingVision president and CEO Stéphane Boissel said: “We are committed to saving sight with pioneering genomics and firmly believe that SPVN06 has the potential to make a meaningful impact in patients’ lives and bring hope to those affected by retinitis pigmentosa.
“This trial is just the beginning; we have a broad pipeline of vision-saving treatments that leverage cutting-edge genomic technologies that will transform the current treatment of retinal disease.
“We look forward to reporting on the progress of SPVN06 and our other programmes as we rapidly move our portfolio through development.”
SPVN06 is a mutation-agnostic, AAV gene therapy comprising a rod-derived cone viability factor and a rod-derived cone viability factor long form.
