SparingVision Presents One Year Results from the PHENOROD2 Natural History Study and Preliminary SPVN06 Safety Data at Key Congresses

• First analysis from PHENOROD2, one of the largest prospective natural history studies in RCD patients due to a genetic variant in RHO, PDE6A, or PDE6B; data presented at ARVO 2024 Annual Meeting
• PRODYGY trial demonstrated favorable initial safety and tolerability with low and medium doses of SPVN06; data presented at ARVO 2024 Annual Meeting and ASGCT 27^th Annual Meeting

Paris, 13 May 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, has presented one year follow-up data from the ongoing four-year prospective natural history study PHENOROD2, which showed a slow but heterogeneous reduction in the area of preserved photoreceptors and the identification of a subset of patients with a higher rate of disease progression using structural markers of clinical interest. In addition, the Company presented initial one-month and six-month safety data from the Phase I/II PRODYGY clinical trial of its lead gene-agnostic investigational gene therapy, SPVN06 demonstrating a favorable safety profile.

The PHENOROD2 study is one of the largest prospective natural history studies ever conducted in patients with rod-cone dystrophy (RCD) due to a variant in the gene RHO, PDE6A, or PDE6B. A cohort of 80 patients was recruited regardless of their disease duration, provided they were not legally blind, as defined by a best-corrected visual acuity (BCVA) of ≥ 20/200 in at least one eye and retained a horizontal diameter of binocular visual field ≥ 5°.

The one-year follow-up data from PHENOROD2 presented at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting, showed — as expected for this patient population — a slow but heterogeneous reduction of the area of preserved photoreceptors, following analysis of structural markers including the horizontal width of the ellipsoid zone (hEZ) and the horizontal and vertical diameters of the inner ring of hyperautofluorescence (hFAF and vFAF). The mean annual loss for these parameters was -7%. Results from the study also identified a subset of patients (47% of the cohort) with a higher rate of disease progression, regarded as ‘Fast Progressors’. The follow-up of PHENOROD2 patients continues, and covariate analyses will be conducted to examine specific patient populations more closely.

Identification of structural markers of accelerated degeneration in certain patients is an important finding for therapies like SPVN06 looking to promote cone survival and slow down disease progression. It is of high interest for the ongoing Phase I/II trial PRODYGY which will assess the safety and preliminary efficacy of SPVN06 at 12 months using the same markers among others. SPVN06 is a breakthrough gene-agnostic gene therapy approach aimed at stopping or slowing disease progression in patients affected by RCD. Exploratory efficacy assessment in the PRODYGY Phase I/II trial will include a descriptive comparison to PHENOROD2 findings.

Initial one-month and six-month data of the first two cohorts of the dose-escalation phase (Part 1) of the Phase I/II PRODYGY clinical trial of SPVN06 in patients with severe RCD, which were first unveiled at the Macula Society 47^th Annual Meeting, were also presented at the ARVO 2024 Annual Meeting and the American Society of Gene and Cell Therapy (ASGCT) 27^th Annual Meeting. Favorable safety and tolerability profiles were observed with the low and medium doses of SPVN06 across six patients (three patients per cohort). Enrollment and treatment administration of the third cohort, comprising three patients at a higher dose, are underway following a positive recommendation to continue the trial without modifications from the independent Data Safety Monitoring Board.  The Company expects to transition to the second part of PRODYGY, a controlled, randomized three-arm study extension, in the second half of 2024.

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, said: “The PHENOROD2 trial results provide greater insights into the progression of rod-cone dystrophies, a heterogeneous group of rare inherited retinal disorders where natural history studies are limited, and unmet medical need remains high for patients. Thanks to the wealth of data collected, we have been able to identify specific structural markers of rapid disease progression for a subset of ‘faster progressors’; this is encouraging as we look to identify safety and preliminary signs of efficacy at 12 months in our ongoing clinical trial, PRODYGY. We look forward to sharing further updates as our research progresses in both PHENOROD2 and PRODYGY.”

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