Final Dose Reached in PRODYGY Trial for RP Gene Therapy

SparingVision, a genomic medicine company specializing in retinal disease treatment, has reported a significant milestone in its Phase I/II clinical trial for SPVN06, an innovative gene therapy for retinitis pigmentosa (RP). The trial has reached the final dose cohort in its dose-escalation phase, following a positive recommendation from the Data Safety Monitoring Board (DSMB). This advancement paves the way for the initiation of the controlled study segment, expected to commence in the second quarter of 2024.

SPVN06 represents a groundbreaking approach to halting or decelerating the progression of rod-cone dystrophy (RCD), a condition that can lead to blindness, irrespective of genetic predisposition. The company is initially targeting mid-stage RP, a prevalent inherited cause of blindness worldwide.

The trial's progression to the highest dose of SPVN06 for the third cohort follows an earlier endorsement from the DSMB to administer the medium dose in August 2023. Both endorsements were based on the favorable safety and tolerability observed in patients who received the initial and medium doses of SPVN06. Upon completion of the final cohort, SparingVision intends to advance to the second phase of the trial, which will involve a controlled, double-masked, randomized extension phase with additional clinical sites in the United States. The primary endpoint of the trial is anticipated to be achieved in the latter half of 2025.

Stéphane Boissel, President and CEO of SparingVision, expressed optimism regarding the safety and tolerability profile demonstrated by SPVN06. He stated that this milestone not only allows for the progression to the highest dose and the subsequent start of the second phase but also bolsters confidence in expanding the evaluation of SPVN06 to geographic atrophy (GA), another significant cause of blindness. Boissel highlighted the company's commitment to advancing its genomic medicine portfolio to preserve sight.

Encouraged by the safety data from the PRODYGY trial and supported by evidence and expert insights, SparingVision sees potential for SPVN06 in treating other retinal diseases where cone preservation is crucial for disease management. The company plans to explore the benefits of its gene therapy for GA, with the aim of initiating Investigational New Drug (IND)-enabling studies in 2024.

The PRODYGY trial is a Phase I/II multicentric study designed to evaluate the safety, tolerability, preliminary efficacy, and quality of life following a single subretinal injection of SPVN06 in adult patients with RCD due to mutations in specific genes. The study aims to recruit 33 patients in two parts: an open-label dose-escalation phase and a controlled, double-masked, randomized extension phase.

SparingVision is a global leader in ophthalmology, offering hope to millions affected by retinal diseases that currently lack viable treatments. The company has developed a range of cutting-edge technologies, including gene therapy and CRISPR, to deliver breakthrough treatments. Both SPVN06 and SPVN20 are gene-agnostic treatments for Retinitis Pigmentosa, a leading cause of blindness globally. SparingVision also collaborates with Intellia Therapeutics to develop novel genome editing treatments using CRISPR-Cas9 technology.

SPVN06 is a proprietary AAV gene therapy that includes a neurotrophic factor and an enzyme to reduce oxidative stress, aiming to slow or stop the degeneration of cone photoreceptors in patients with RCD. The therapy has the potential to be applied to a variety of diseases where rod loss is an early indicator of the condition. SparingVision, a spin-off from the Paris Vision Institute, is backed by international investors and is committed to developing treatments for RP and dry Age-related Macular Degeneration (AMD), conditions that currently have no approved treatments.