SparingVision Reaches Final Dose Escalation Step in PRODYGY Trial with SPVN06 for retinitis pigmentosa

SparingVision Reaches Final Dose Escalation Step in PRODYGY Trial with SPVN06 for retinitis pigmentosa

• Positive recommendation received from the Data Safety Monitoring Board (DSMB) enabling progression to highest dose of Part 1 of the PRODYGY trial
• Favorable safety and tolerability profiles observed with first two SPVN06 doses across six patients (three patients per cohort)
• Exploration of SPVN06 in geographic atrophy to begin in 2024

Paris, 24 January 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces a key advancement in its Phase I/II clinical trial (PRODYGY, NCT05748873) for SPVN06, the Company’s lead gene-agnostic investigational gene therapy for the treatment of retinitis pigmentosa (RP). The trial has now progressed to the final dose cohort of the dose-escalation phase (Part 1). This positive recommendation from the Data Safety Monitoring Board (DSMB) represents a further step towards the commencement of the controlled part of the study (Part 2) anticipated to start in Q2 2024, in line with study development timelines.

SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background. SparingVision is initially focusing on mid-stage RP, one of the leading inherited causes of blindness globally.

This progress of the PRODYGY trial to the third cohort at the highest dose of SPVN06 follows an earlier positive recommendation from the DSMB to dose the second cohort at the medium dose in August 2023. The two positive recommendations received to date from the DSMB were based on the favorable safety and tolerability profiles observed in patients treated with the lower (first cohort) and medium (second cohort) doses of SPVN06. After completion of the third and final cohort in Part 1, the Company plans to advance the PRODYGY trial to Part 2, a three-arm controlled, double-masked, randomized extension phase investigating two doses of SPVN06 and one untreated arm, with additional clinical sites opening in the United States. The primary endpoint of the PRODYGY Phase I/II trial is expected to be reached in the second half of 2025, twelve months after the last patient in Part 2 is dosed.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “We are encouraged by the strong safety and tolerability profile of SPVN06 demonstrated so far. This milestone not only allows us to progress to the highest dose and, pending further DSMB positive recommendation, start Part 2 in Q2 2024; it also gives us the added confidence to expand the evaluation of SPVN06 to geographic atrophy (GA), another significant blinding condition. We are excited about the future as we continue to advance our portfolio of pioneering genomic medicines to save sight.”

With the encouraging safety data gathered in PRODYGY so far and supported by a body of evidence and KOL insights, SparingVision believes in the strong potential of SPVN06 in other retinal diseases for which cone preservation is the landmark for stopping disease progression. The Company intends to explore the benefits of its gene therapy for the treatment of geographic atrophy (GA), with a view to potentially initiating Investigational New Drug (IND)-enabling studies for GA in 2024.

PRODYGY trial

PRODYGY (Promising ROd-cone DYstrophy Gene therapY) is a multicentric Phase I/II trial to assess the safety, tolerability as well as preliminary efficacy and quality-of-life following a single subretinal injection of SPVN06 in the worst-seeing eye of adult patients with RCD due to a mutation in the RHO, PDE6A, or PDE6B gene. The study aims to recruit a total of 33 patients in two parts:

• Part 1: open-label, dose-escalation phase including three cohorts of three patients with severe advanced RCD, to determine two recommended best-tolerated doses for Part Two.
• Part 2: controlled, double-masked, randomized extension phase including 24 patients with intermediate advanced RP, divided into three cohorts: six untreated subjects and 18 patients receiving one of the two recommended doses determined in Part One.

Further information on the trial can be found on www.ClinicalTrials.gov (CT identifier: NCT05748873).

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About SparingVision

SparingVision is a global ophthalmology leader bringing new hope to millions affected by retinal diseases, for which there are currently no viable treatments. The Company has assembled a suite of cutting-edge technologies from gene therapy to CRISPR, enabling it to deploy the right technology to the right disease and ensure the delivery of breakthrough treatments to millions of patients.

Both of its products, SPVN06 and SPVN20 go beyond single gene correction therapies to deliver new gene-agnostic treatments for Retinitis Pigmentosa, a group of inherited retinal diseases which are the leading cause of blindness globally. The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ:NTLA) to develop novel genome editing-based treatments for ocular disease utilizing CRISPR-Cas9 technology.

SparingVision is a spin-off from the Paris Vision Institute and backed by high-quality international investors including 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital and Ysios Capital.
Visit www.sparingvision.com for more and follow us on LinkedIn and X (formerly Twitter) @SparingVision

About SPVN06

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor (Rod derived Cone Viability Factor, RdCVF) and one enzyme reducing oxidative stress (Rod derived Cone Viability Factor Long form, RdCVFL). Acting synergistically, RdCVF and RdCVFL aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVision’s primary disease targets are retinitis pigmentosa (RP), one of the most common inherited retinal diseases that affects an estimated two million patients worldwide and dry Age-related Macular Degeneration (AMD). There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases, where the loss of rods is known to be an early signal of the disease. SPVN06 is the result of decades of world-leading ophthalmology research by SparingVision founders José-Alain Sahel and Thierry Léveillard at the Paris Vision Institute