SPVN-06

SparingVision Reaches Final Dose Escalation Step in PRODYGY Trial with SPVN06 for retinitis pigmentosa Positive recommendation received from the Data Safety Monitoring Board (DSMB) enabling progression to highest dose of Part 1 of the PRODYGY trialFavorable safety and tolerability profiles observed with first two SPVN06 doses across six patients (three patients per cohort)Exploration of SPVN06 in geographic atrophy to begin in 2024 Paris, 24 January 2024 – SparingVision (“the Company”), a clinical-stage genomic medicine company transforming the treatment of retinal disease, today announces a key advancement in its Phase I/II clinical trial (PRODYGY, NCT05748873) for SPVN06, the Company’s lead gene-agnostic investigational gene therapy for the treatment of retinitis pigmentosa (RP). The trial has now progressed to the final dose cohort of the dose-escalation phase (Part 1). This positive recommendation from the Data Safety Monitoring Board (DSMB) represents a further step towards the commencement of the controlled part of the study (Part 2) anticipated to start in Q2 2024, in line with study development timelines. SPVN06 is a breakthrough gene therapy approach aimed at stopping or slowing disease progression in patients affected by rod-cone dystrophy, (RCD), regardless of their genetic background. SparingVision is initially focusing on mid-stage RP, one of the leading inherited causes of blindness globally. This progress of the PRODYGY trial to the third cohort at the highest dose of SPVN06 follows an earlier positive recommendation from the DSMB to dose the second cohort at the medium dose in August 2023. The two positive recommendations received to date from the DSMB were based on the favorable safety and tolerability profiles observed in patients treated with the lower (first cohort) and medium (second cohort) doses of SPVN06. After completion of the third and final cohort in Part 1, the Company plans to advance the PRODYGY trial to Part 2, a three-arm controlled, double-masked, randomized extension phase investigating two doses of SPVN06 and one untreated arm, with additional clinical sites opening in the United States. The primary endpoint of the PRODYGY Phase I/II trial is expected to be reached in the second half of 2025, twelve months after the last patient in Part 2 is dosed. Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “We are encouraged by the strong safety and tolerability profile of SPVN06 demonstrated so far. This milestone not only allows us to progress to the highest dose and, pending further DSMB positive recommendation, start Part 2 in Q2 2024; it also gives us the added confidence to expand the evaluation of SPVN06 to geographic atrophy (GA), another significant blinding condition. We are excited about the future as we continue to advance our portfolio of pioneering genomic medicines to save sight.” With the encouraging safety data gathered in PRODYGY so far and supported by a body of evidence and KOL insights, SparingVision believes in the strong potential of SPVN06 in other retinal diseases for which cone preservation is the landmark for stopping disease progression. The Company intends to explore the benefits of its gene therapy for the treatment of geographic atrophy (GA), with a view to potentially initiating Investigational New Drug (IND)-enabling studies for GA in 2024. 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. The study aims to recruit a total of 33 patients in two parts: Part 1: open-label, dose-escalation phase including three cohorts of three patients with severe advanced RCD, to determine two recommended best-tolerated doses for Part Two.Part 2: controlled, double-masked, randomized extension phase including 24 patients with intermediate advanced RP, divided into three cohorts: six untreated subjects and 18 patients receiving one of the two recommended doses determined in Part One. Further information on the trial can be found on www.ClinicalTrials.gov (CT identifier: NCT05748873). **ENDS** Contacts: SparingVisionStéphane BoisselPresident and CEONathalie TrepoInvestor Relations nathalie.trepo@sparingvision.comICR ConsiliumAmber Fennell, Namrata Taak, Davide Salvi+44 (0)20 3709 5700sparingvision@consilium-comms.com 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 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 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

Adam Zamecnik @a_zamecnik SparingVision plans to start a Phase I trial of its eye disease gene therapy SPVN20 in H2 2024. Image Credit: Shutterstock / Svetazi SparingVision expects to start a new Phase I trial of its ocular gene therapy SPVN20 in H2 2024, said CEO Stéphane Boissel in an interview with Pharmaceutical Technology . The French company is finalising clinical trial application (CTA)-enabling studies for its gene therapy, with plans to submit the CTA in 2024, added Boissel. Recommended Reports Reports LOA and PTSR Model - Gene Therapy to Target BCMA for Relapsed and Refractory Multiple Myeloma GlobalData Reports LOA and PTSR Model - Gene Therapy for Relapsed and Refractory Multiple Myeloma GlobalData View all Companies Intelligence SparingVision SAS CTA Inc Boissel Gamut Therapeutics SAS View all SPVN20 is an adeno-associated virus-based gene therapy that could improve visual acuity in advanced and late-stage retinitis pigmentosa patients, as per the company. The gene therapy seeks to restore the function of viable non-functional cones by encoding for a variant of GIRK, a GPCR-activated ion channel that reactivates cone function, per an April 2021 press release. In April 2021, SparingVision acquired the biotech GAMUT Therapeutics, which also gave it access to the company’s lead candidate, which was renamed SPVN20. The Phase I trial, named NYRVANA, will have a data readout in 2025, said Boissel. The trial will be shorter and smaller than the ongoing Phase I/II PRODYGY study (NCT05748873) of another therapy SPVN06 in stage two and three retinitis pigmentosa, explained Boissel. Although Boissel did not disclose the exact number of subjects in the trial, he said dose escalation trials usually investigate between two to three doses with three to five subjects per dose. According to the National Eye Institute, retinitis pigmentosa is a group of rare eye disorders , which result in a gradual breakdown of retina cells, rod-cone dystrophy and vision loss. There is no available cure for the condition. Stéphane Boissel, CEO of SparingVision Last month, SparingVision shared positive initial safety data from the PRODYGY study’s first cohort, stating that the therapy was well-tolerated at low doses in the first cohort. SPVN06 is an adeno-associated virus-based gene therapy that consists of one neurotrophic factor RdCVF and one enzyme that slows the degeneration of cone photoreceptors that lead to blindness in patients with rod-cone dystrophies, based on the company’s website. In June 2020, the European Commission granted SPVN06 orphan drug status in inherited retinal dystrophies. The company is now working on recruiting patients for further cohorts in PRODYGY. SparingVision expects to recruit subjects for the second cohort in early November, said Boissel. Additionally, the enrollment of participants for the third cohort will finish sometime early next year, added Boissel. Afterwards, the company will move into the second part of the trial, which will be a control study that will recruit patients between Q1 and Q3 2024, the CEO stated. As part of this, the company expects to open up three more US-based sites, bringing the total to five trial locations, he said. SparingVision expects to have results ready from the trial in 2025, added Boissel. SparingVision expects that its current funding secured through a past Series B will last until the second half of 2025, he added. In September 2022, the company raised €75m as part of the Series B. C ell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Cell and gene therapies: Pipe dream to pipeline The cell and gene industry is gaining momentum, with a new wave of therapies promising to transform the way doctors treat, and even cure, disease. In this report, Cytiva and GlobalData have collaborated to explore the rise of the cell and gene therapy industries, the current state of the market, present and future opportunities for advancement, and the challenges that lie ahead. By Cytiva Thematic By downloading this case study, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy