SPVN-06

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

SparingVision Reports Positive Initial Safety Data from the first cohort treated in its PRODYGY Phase I/II Gene Therapy Trial Lead gene therapy asset, SPVN06, reported to be well-tolerated in first dosed cohort at low dose with a favorable safety profilePositive recommendation received from Data Safety Monitoring Board (DSMB) to dose second cohort at the medium dose Paris, August 30, 2023 – SparingVision (“the Company”), a clinical-stage genomic medicine company developing vision-saving treatments for ocular diseases, today announced positive initial safety data from the Phase I/II clinical trial (‘PRODYGY’, NCT05748873) of SPVN06, the Company’s lead gene-agnostic gene therapy for the treatment of retinitis pigmentosa (RP). 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. Safety data collected on the first three patients (cohort 1) treated with a low dose showed that SPVN06 has been well tolerated with a favorable safety profile. The patients were each injected at the 15-20 National Hospital in Paris, France. Following review of the data, the Data Safety Monitoring Board (DSMB) concluded that it is safe to initiate the second cohort at a medium dose. Stéphane Boissel, President and Chief Executive Officer of SparingVision, said: “We are committed to saving sight with pioneering genomics and firmly believe that SPVN06 has the potential to make a meaningful impact in patients' lives and bring hope to those affected by retinitis pigmentosa. This trial is just the beginning; we have a broad pipeline of vision-saving treatments that leverage cutting edge genomic technologies that will transform current treatment of retinal disease. We look forward to reporting on the progress of SPVN06 and our other programs as we rapidly move our portfolio through development.” Avril Daly, Chief Executive Officer of Retina International, a patient advocacy group, commented: “The positive initial safety data with SPVN06 is an important step forward for all those living with retinitis pigmentosa. As a community, we have highlighted the need for a therapy that addresses the wide range of genetic backgrounds that can cause RP for many years now. SparingVision's approach with SPVN06 offers hope for those of us who have been impacted by this degenerative blinding disease. We are eagerly awaiting the results of this Phase I/II trial and the potential for this therapy to slow or stop the progression of RP." The primary endpoint of the PRODYGY Phase I/II trial is expected to be reached in 2025 and preliminary efficacy results are expected within the same year. PRODYGY trial PRODYGY 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 will recruit a total of 33 patients, in two steps: Step 1: open-label, dose-escalation phase including three cohorts of three patients with severe advanced RCD, to determine two recommended best-tolerated doses for Step Two.Step 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 Step One. The principal investigators of the trial are Pr. Isabelle Audo at the 15-20 National Hospital in Paris, France and Dr. Joseph Martel at the Eye Center of the University of Pittsburgh Medical Center, Pittsburgh, USA. Further details of the trial can be found on clinicaltrials.gov under the identifier NCT05748873. SPVN06SPVN06 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.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. **ENDS** Contacts: SparingVisionStéphane BoisselPresident and CEONathalie TrepoInvestor Relations nathalie.trepo@sparingvision.comConsilium Strategic CommunicationsAmber Fennell, Namrata Taak, Davide Salvi+44 (0)20 3709 5700sparingvision@consilium-comms.com NOTES TO EDITORS: About SparingVisionSparingVision is a clinical-stage 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 gene-independent 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 a spin-off from the Paris Vision Institute and is backed by high-quality international investors including 4BIO Capital, AdBio Partners, Bpifrance, Fondation Voir & Entendre, Intellia Therapeutics, Jeito Capital, RD Fund (US), UPMC Enterprises, and Ysios Capital. Visit www.sparingvision.com for more and follow us on LinkedIn and Twitter @SparingVision. About Inherited Retinal DiseasesInherited retinal diseases are a group of progressive eye conditions that can cause severe vision loss and, in certain cases, lead to total blindness. One of the most common examples is Retinitis Pigmentosa, a type of retinal dystrophy that involves a breakdown and loss of cells in the retina. Retinal dystrophies are caused by mutations in any one of more than 270 genes identified to date (over 80 genes for RP alone) and have become a target for novel genomic medicines.