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September, 23 2024 • Articles

SparingVision Reports Progress on SPVN06 at Major Ophthalmology Conferences

SPVN06 demonstrates continued favorable safety profile at 12 months for low dose and 6 months for medium dose in PRODYGY Phase I/II trial

Data presented at the Retina Society Annual Scientific Meeting (Lisbon, September 11) and Euretina Congress (Barcelona, September 21)

Paris, 23 September 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, is pleased to announce the presentation of 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).

The data, which includes 12-month safety results for the low dose cohort and 6-month safety results for the medium dose cohort, demonstrates a continued favorable safety profile. This builds upon the previously announced initial six-month and one-month data from the first two cohorts of the dose-escalation phase (Part 1) of PRODYGY, which showed favorable safety and tolerability profiles.

The results were shared by Dr. Joseph Martel, Principal Investigator of the trial in the U.S., in an oral presentation at the Retina Society Annual Scientific Meeting in Lisbon, Portugal as well as by Professor Isabelle Audo, Principal Investigator of the study in France at Euretina Congress in Barcelona, Spain.

Enrollment and treatment administration of the third cohort, comprising three patients at a higher dose of SPVN06, are currently underway.

Additionally, SparingVision has presented, at Euretina Congress, the one-year follow-up data from the ongoing PHENOROD2 natural history study, initially announced in May 2024. These data revealed important patterns in disease progression among RP patients, including the identification of a subset of patients with accelerated degeneration. These findings provide SparingVision with valuable insights that will inform the development and assessment of therapies like SPVN06.

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, said: "The extended safety data from our PRODYGY trial continues to be encouraging, reinforcing the robust safety profile of SPVN06 observed to date. As we progress to the highest dose, these results provide a solid foundation for the continued development of our gene-agnostic therapy for retinitis pigmentosa. The lack of serious adverse events or dose-limiting toxicities across multiple timepoints is particularly noteworthy.

Crucially, our PHENOROD2 natural history study has uncovered key markers of disease progression that are directly informing our SPVN06 development strategy. The identification of structural markers of accelerated degeneration in certain patients is a significant finding for therapies like SPVN06 that aim to promote cone survival and slow down disease progression.

This interplay between PHENOROD2 and PRODYGY is enhancing our understanding of both disease progression and the potential impact of SPVN06. These findings continue to encourage our belief in SPVN06's potential to make a meaningful difference for patients with inherited retinal diseases."

**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 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 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).