SparingVision announced positive initial safety data from the Phase I/II clinical trial (referred to as PRODYGY) evaluating SPVN06, the company's primary gene-agnostic gene therapy designed for retinitis pigmentosa (RP).
SPVN06 is an innovative gene therapy approach intended to halt or slow the progression of the disease in individuals affected by rod-cone dystrophy (RCD), irrespective of their genetic makeup.
The safety data gathered from the first three patients (forming cohort 1), who received a low dosage of SPVN06, indicate that the therapy has been well tolerated and exhibits a favorable safety profile. The injections were administered at the 15-20 National Hospital in Paris, France. Following a thorough examination of the data, the Data Safety Monitoring Board (DSMB) has affirmed the safety of proceeding to the second cohort with a medium dose.
Stéphane Boissel, President and Chief Executive Officer of SparingVision, 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 current treatment of retinal disease. We look forward to reporting on the progress of SPVN06 and our other programs as we rapidly move our portfolio through development.”
The PRODYGY Phase I/II trial's primary endpoint is anticipated to be reached in 2025, with preliminary efficacy outcomes expected during the same year.
PRODYGY is a Phase I/II multicentric trial designed to evaluate the safety, tolerability, preliminary efficacy, and quality-of-life outcomes following a single subretinal injection of SPVN06 in the worse-seeing eye of adult patients diagnosed with RCD due to a mutation in the RHO, PDE6A, or PDE6B gene. The trial will enroll a total of 33 patients through two distinct phases:
● Phase 1: This is an open-label, dose-escalation phase comprising three cohorts, each consisting of three patients with severe advanced RCD. Its purpose is to determine the two recommended best-tolerated doses for Phase 2.
● Phase 2: This phase is a controlled, double-masked, randomized extension phase and will include 24 patients with intermediate advanced RP, divided into three cohorts. Six subjects will remain untreated, while the remaining 18 patients will receive one of the two Recommended Doses established in Phase 1.
The lead investigators overseeing this trial are Pr. Isabelle Audo at the 15-20 National Hospital in Paris, France, and Dr. Joseph Martel at the Eye Center of the University of Pittsburgh Medical Center in Pittsburgh, USA.