Paris, May 5, 2022 – SparingVision (“the Company”), a genomic medicine company developing vision saving treatments for ocular diseases, announces today that it has presented progress of its lead gene therapy program, SPVN06 in an oral presentation and two posters at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meeting.

SparingVision first presented pharmacology data on SPVN06, a breakthrough gene therapy approach targeting Inherited Retinal Diseases (IRDs), demonstrating a highly significant protection of visual function as well as greater cone density following subretinal administration of SPVN06 in a murine model. The Company is currently conducting a GLP[1] toxicology and biodistribution study which will mark the last step in preclinical validation ahead of the Company’s Clinical Trial Authorisation (CTA) submission and Investigational New Drug (IND) application which are both expected in Q3 this year.

Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “The positive progress we are seeing in our preclinical data is extremely promising as we head towards CTA and IND submission. We are also honoured that ARVO has given us the platform to share our findings regarding the natural history of retinitis pigmentosa in an oral presentation; this is a true testament to the significance of our research and recognizes our position as a pioneer and thought leader in research on mutation-agnostic and disease-independent gene therapy in the ocular space.” 

SparingVision later provided updates on its two natural history studies conducted in parallel to the IND-enabling program for SPVN06: PHENOROD1 and PHENOROD2. Together, these natural history studies provide greater insight into the course of development of retinitis pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide, and the primary target of SPVN06. They are expected to inform the Company on the target patient population and potential efficacy endpoints of the upcoming first-in-man clinical trial with SPVN06, named PRODYGY. To some extent, they will be used as a lead-in study for the PRODYGY study.

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, added, “Despite retinitis pigmentosa being the leading cause of blindness worldwide, there is a great need for clinical information on the disorder’s evolution. As such, there remains a strong interest in natural history studies in the field. The data from our PHENOROD1 and PHERNOROD2 studies offer novel insights to inform clinical trial design and treatment development that will prove invaluable as we move towards the clinic.”

PHENOROD1 is a retrospective study of RP due to RHO, PDE6A, or PDE6B mutations, the purpose of which was to better understand the natural history of rod-cone degeneration in RP according to underlying genotypes. PHENOROD2 is a prospective natural history study of RP due to RHO, PDE6A, or PDE6B mutations which looked at the evolution of RP.

More details of the presentations can be found below:

Oral Presentation: Prospective natural history of retinitis pigmentosa due to RHO, PDE6A, or PDE6B mutations: interim analysis of the PHENOROD2 study (Presentation #4294).

Presenter: Anne Celle, Clinical Project Manager at SparingVision

• Interim analysis of the first 44 patients enrolled showed that overall, visual acuity remained stable at one year of follow-up. Kinetic visual fields showed similar results, in this first half of the PHENOROD2 cohort including less affected patients. Future analyses including the entire PHENOROD2 cohort may show a more defined picture, with additional functional and structural measures such as microperimetry and ellipsoid zone. Patient recruitment in the PHENOROD 2 study has now been completed (n=82).

Poster Presentation: Retrospective natural history of retinitis pigmentosa due to RHO, PDE6A, or PDE6B mutations: the PHENOROD1 study (Poster #4497-F0284).

Presenter: Alice Le Meur, Director, Clinical Projects at SparingVision

• Analysis of functional and structural visual parameters allowed to better define the natural history of rod-cone dystrophy and reported the slow progression of visual loss in 110 patients with RP across all three mutations over an average of 6 years of follow-up. Non-parametric regression models further demonstrated progressive degeneration, as shown by the evolution of visual functional parameters Best-Corrected Visual Acuity (BCVA) and Visual Field (VF); however, the rates of change varied over several decades with onset ofvisual field constriction generally occurring earlier, followed by visual acuity loss later in life. These results are of particular interest in optimizing the design of clinical studies and the selection of patients in the development of treatments aimed at protecting photoreceptors, such as SPVN06.

Poster Presentation: SPVN06, a novel mutation-independent AAV-based gene therapy, dramatically reduces vision loss in the rd10 mouse model of rod-cone dystrophy (Poster #56 – A0029).

Presenter: Dr. Florence Lorget, PharmD, PhD, DABT, Chief Development Sciences Officer at SparingVision

• SPVN06 pharmacological activity was demonstrated in an autosomal recessive model of retinitis pigmentosa (rd10 mouse model). SPVN06 subretinal administration at P18 (1E8 vg/eye, 1 µL) resulted in a highly significant slow-down in visual acuity loss measured by optokinetic at all evaluated timepoints (P32, P38 and P45) and greater cone density at P48 when compared to controls.

[1] GLP : Good Laboratory Practices

NOTES TO EDITORS:

About SparingVision

SparingVision is a genomic medicines company with a mission to translate pioneering science into vision saving treatments. Leveraging its unparalleled understanding of retinal diseases, SparingVision has built the world’s most compelling portfolio of synergistic cutting-edge gene therapy and genome editing treatments for inherited retinal diseases (IRDs). Both of its most advanced products, SPVN06 and SPVN20 look to go beyond single gene correction therapies to deliver new mutation agnostic treatments for Retinitis Pigmentosa (RP), a group of IRDs 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 diseases utilizing CRISPR-Cas9 technology.

SparingVision is 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, Ysios Capital.

Visit www.sparingvision.com for more and follow us on LinkedIn and Twitter @SparingVision

About SPVN06:

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, 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 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. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2022.