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October, 24 2024 • Articles

SparingVision Presents Updates on PRODYGY Trial and PHENOROD2 Study at ESGCT 2024

First oral presentation on PRODYGY at a major cell and gene therapy-focused conference

Completion of PRODYGY Step 1 (9 patients treated across three doses through dose escalation) 

Paris, 24 October 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces the presentation of a progress update from the Phase I/II PRODYGY clinical trial of its lead gene-agnostic investigational gene therapy program, SPVN06, at the European Society of Gene & Cell Therapy (ESGCT) 31^st Annual Congress in Rome, Italy.

Positive longer-term safety data from the Phase I/II PRODYGY clinical trial of its lead investigational therapy, SPVN06, for the treatment of retinitis pigmentosa (RP) was shared in an oral presentation by Laure Blouin, Director, Clinical Science and Medical Communications at SparingVision. These data, previously announced in September 2024, show that SPVN06 demonstrates a favorable safety profile at 12 months for the low dose cohort and 6 months for the medium dose cohort in the PRODYGY Phase I/II trial.

The Company also provided a recruitment update on the PRODYGY trial, announcing that enrolment of patients in the dose-escalation phase (Step 1) is now complete. This part aimed to evaluate the safety and tolerability of SPVN06 at different dose levels. A total of 9 patients were dosed since Q2 2023 in Step 1. SparingVision plans to initiate the controlled, randomized extension phase (Step 2) in Q4 2024, pending approval from the Data Safety and Monitoring Board (DSMB) which will review all safety data collected to date and recommend two doses for testing in the next part of the trial. A total of 24 patients should be enrolled in Step 2 across 3 arms, including one control arm of untreated patients, across 5 or 6 specialised clinical centers in France and the USA.

At ESGCT, SparingVision also presented one-year follow-up data from the ongoing PHENOROD2 prospective natural history study, in a poster presentation by Alice Le Meur, Head of Clinical Operations for Gene Therapies at SparingVision. These data, initially announced in May 2024, revealed important patterns in disease progression among patients with RP, including the identification of a subset of patients with accelerated degeneration.

Additionally, Fabien Dorange, Director of Analytical Sciences at SparingVision, presented a poster on a novel analytical method developed by the Company. This method, a triplex digital PCR (dPCR) assay, is designed to detect and quantify replicant-competent AAV genomes (rcAAVs) in AAV-based gene therapy products. The study showed that this new assay could complement existing safety testing methods, potentially enhancing the quality control process for AAV-based gene therapies like SPVN06.

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, said: “As highlighted in our first oral presentation on PRODYGY at a major gene therapy-focused conference, the significant progress in the ongoing clinical trial underscores our confidence in SPVN06's potential to transform the treatment landscape for retinitis pigmentosa. Our presentations on PHENOROD2 and our novel triplex dPCR assay further demonstrate our commitment to deepening our understanding of retinal disease progression, pioneering new approaches, and setting new benchmarks for safety in gene therapy development.”

**ENDS**

Contacts:

SparingVision
Stéphane Boissel	President and CEO
Nathalie Trepo	Investor Relations nathalie.trepo@sparingvision.com
ICR Consilium
Amber Fennell, Namrata Taak, Davide Salvi	+44 (0)20 3709 5700 sparingvision@consilium-comms.com

 

DISCLAIMER
Dr. Jose-Alain Sahel and UPMC have financial interests in the study sponsor, SparingVision. These financial interests mean it is possible that the results of this research could lead to personal profit for Dr. Sahel and to institutional profit for UPMC. The conflicts of interest presented by these relationships are being managed by the University and UPMC.

NOTES TO EDITORS:

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 investigational 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 one of the leading causes 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 vector based investigational 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.

About PHENOROD2
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 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 investigational 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.  

Further information on the PHENOROD2 trial can be found on www.ClinicalTrials.gov (CT identifier: NCT04285398).

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