No serious adverse events in the first two cohorts of patients dosed with SPVN06 (N=6)

Observed ocular adverse events predominantly procedural, transient and resolved

SparingVision founder, José-Alain Sahel, MD, awarded the Macula Society Michaelson Award

Paris, 8 February 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, has presented positive initial safety data from the Phase I/II PRODYGY clinical trial of its lead gene-agnostic investigational gene therapy, SPVN06, for the treatment of retinitis pigmentosa (RP). Data from six patients treated with SPVN06 were shared by Professor Isabelle Audo^[1],[2], Principal Investigator of the study, in an oral presentation at the Macula Society 47^th Annual Meeting being held February 7-10, 2024 in Palm Springs, USA.

Initial six-month data on the three patients who received a low dose of SPVN06 showed no serious adverse events, significant inflammation, discontinuation, or dose-limiting toxicity. Additionally, one-month data on the three patients treated with a medium dose corroborated these findings.

Ocular adverse events observed were primarily associated with procedural aspects and resolved swiftly.

Enrollment and treatment of the third cohort, comprising three patients at a higher dose, are underway following a favorable review and positive recommendation from the Data Safety Monitoring Board.

Dr Daniel C. Chung, Chief Medical Officer of SparingVision, said: “The lack of serious adverse effects, discontinuations, or dose-limiting toxicity and no significant inflammation or immune response demonstrated in the preliminary safety data for SPVN06 in our early groups is highly encouraging. This robust safety profile bolsters our confidence as we move forward with the study.”

SPVN06 is a breakthrough gene-agnostic gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy (RCD). SparingVision is initially focusing on mid-stage RP, the most common form of RCD and one of the leading inherited causes of blindness globally. The Company aims to investigate the advantages of its gene therapy in treating geographic atrophy (GA) and potentially commence Investigational New Drug (IND)-enabling studies for GA in 2024.

In addition, SparingVision is delighted to announce that its founder, José-Alain Sahel³, MD, has received the Macula Society Michaelson Award, a distinction presented every three years to an outstanding scientist. Pr. Sahel also delivered the Michaelson lecture on gene-independent strategies for retinal degenerations and organized a related symposium at the meeting.

PRODYGY trial

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).

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.

[1] Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, Centre de référence maladies rares REFERET, INSERM-DHOS CIC 1423, Paris, France

[2] Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France

³ Distinguished professor and chairman of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and director of the UPMC Vision Institute