SparingVision Presents Progress of its Lead Gene Therapy Program, SPVN06, at ARVO 2022

SparingVision Presents Progress of its Lead Gene Therapy Program, SPVN06, at ARVO 2022

Paris, May 5, 2022 – SparingVision (“the Company”), a genomic medicine company developing vision saving treatments for ocular diseases, 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 a 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