SparingVision advances PRODYGY trial into Phase II following positive DSMB recommendation
- DSMB confirms favorable safety profile of SPVN06 across all three doses in patients with severe advanced rod-cone dystrophy (RCD)(n=9)
- Trial to progress to phase II to evaluate the two highest doses of SPVN06 in patients with intermediate advanced RCD
Paris, 21 January, 2025 – SparingVision (“the Company”), announces that following review of the safety data from the dose-escalation phase (Step 1), the independent Data Safety Monitoring Board (DSMB) has recommended continuation to the extension phase (Step 2) of the Phase I/II PRODYGY trial. This extension phase, a phase II, will evaluate the medium and high doses of the Company’s lead gene-agnostic investigational gene therapy product, SPVN06, in 24 patients with advanced intermediate rod-cone dystrophy (RCD).
Safety data from the third and final cohort of Step 1, injected with high-dose SPVN06, demonstrated favorable tolerability. The patients were injected at the 15-20 National Hospital in Paris, France and at the Casey Eye Institute in Portland, Oregon, USA. In total, nine patients have now been injected across three dose levels in Step 1 of the trial.
The extension phase of PRODYGY (Step 2) is designed as a Phase II, controlled, randomized study that will enroll a total of 24 patients across three arms: high dose (n=9), medium dose (n=9) and one control arm of uninjected patients (n=6). This phase will be conducted across six specialized clinical centers in the USA and France. Step 2 will focus on patients with intermediate RCD, specifically those with visual acuity comprised between 20/200 and 20/40 and a visual field equal to or less than 20 degrees.
Stéphane Boissel, CEO of SparingVision, said: “Following the DSMB’s positive recommendation, we are advancing SPVN06 into phase II development, which is an exciting way to start 2025. As an AAV-based vision preservation gene therapy, SPVN06 addresses significant unmet needs in prevalent retinal diseases, regardless of genetic cause. Completion of enrollment of the PRODYGY study and start of the first-in-human study of our second asset, SPVN20, a vision restoration gene therapy program, makes 2025 a transformative year for SparingVision”.
**ENDS**
DISCLAIMER
Dr. Jose-Alain Sahel and UPMC have a financial interest in SparingVision.
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 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, AdBio Partners, 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 target is retinitis pigmentosa (RP), one of the most common inherited retinal diseases that affects an estimated two million patients worldwide. 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, notably Geographic Atrophy (GA) secondary to dry Age-related Macular Degeneration (AMD). SPVN06 is the result of world-leading ophthalmology research by SparingVision founders José-Alain Sahel and Thierry Léveillard at the Paris Vision Institute.
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).