National Eye 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

PHILADELPHIA--(BUSINESS WIRE)-- The National Disease Research Interchange (NDRI) has been awarded $3.7M by the National Institutes of Health (NIH) to support continued national leadership with the recovery and distribution of human organs and tissues for medical research. This new three-year award for NDRI’s Human Tissue and Organs for Research Resource (HTORR) Program parent grant extends the NIH’s ongoing funding support for HTORR to 35 consecutive years. The HTORR Program supports investigators across the full spectrum of research with access to normal and diseased human biospecimens. The parent award is funded by the NIH Office of the Director, Office of Research Infrastructure Programs (ORIP) and receives additional co-funding from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institute of Allergy and Infectious Diseases (NIAID), National Eye Institute (NEI), National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), and the National Heart, Lung, and Blood Institute (NHLBI). “NDRI is grateful to the NIH for the support of our expanding HTORR program,” said Bill Leinweber, President and CEO of NDRI. “It is a privilege for NDRI to build on our long partnership with the NIH as a resource for human biospecimens to meet the evolving needs of scientists.” Through the HTORR program, NDRI provides NIH-funded investigators with donated normal and diseased human biospecimens for their research. This grant renewal includes multiple enhancements such as a new Pilot Program, Pathology Service Options, and a Pathology Consultant Committee. “The enhancements establish a new foundation that will bolster HTORR’s capabilities to meet the emerging needs of the present and next generation of groundbreaking scientific studies,” said Thomas Bell, MS PhD, NDRI’s Chief Scientific Officer and principal investigator for the HTORR grant. “Encompassing additional service and support options along with a new mechanism to incorporate more underrepresented and junior investigators into the program demonstrate the NIH and NDRI’s commitments to sustain HTORR as a leading research resource for the scientific community.” About NDRI The National Disease Research Interchange (NDRI) is the nation’s leading source of human tissues, cells and organs for scientific research. A not-for-profit 501 (c) (3) organization founded in 1980, NDRI is funded in part by the National Institutes of Health, public and private foundations and organizations, pharmaceutical and biotechnology corporations. NDRI is a 24/7 operation that partners with a nationwide network of over 130 tissue source sites (TSS), including organ procurement organizations (OPOs), tissue banks, eye banks, and hospitals. The TSS, are distributed throughout the USA, in 45 states, with concentrations in major metropolitan areas on both the east and west coasts. Their wide geographic distribution allows NDRI to provide biospecimens from donor populations with diverse demographics and facilitates the timely and efficient provision of fresh tissues directly to researchers across the U.S. and around the world. By serving as the liaison between procurement sources and the research community, NDRI is uniquely positioned to support breakthrough advances and discoveries that can affect advances in the treatment and cure of human diseases. View source version on businesswire.com: Contacts Media Inquiries: Matthew Reece Director, Marketing and Communications 215-557-7361, ext. 239 Mreece@ndriresource.org Source: National Disease Research Interchange (NDRI) View this news release online at:

The FDA found several problems with Outlook Therapeutics' application for its bevacizumab reformulation despite the drug's positive pivotal trial. Even without an FDA approval, Roche’s cancer drug Avastin is widely used by doctors to treat certain eye diseases. Outlook Therapeutics has been trying to get an official approval for a reformulated version, but it instead has received a rejection letter. The FDA rejected Outlook’s Lytenava, or bevacizumab-vikg, for the treatment of wet age-related macular degeneration (AMD), the New Jersey company said Wednesday. The drug would have been the first officially labeled, ophthalmic formulation of bevacizumab to enter the market. An approval was largely expected by market watchers given existing knowledge and the long history of off-label use of Avastin in eye diseases. Instead, the FDA found several problems with Outlook’s application despite the package boasting a positive pivotal trial. In the complete response letter to Outlook, the FDA flagged several chemistry, manufacturing and controls (CMC) issues, observations from pre-approval manufacturing inspections, and perhaps most damning, the need for further confirmatory clinical evidence, according to the company. Outlook will request a meeting with the FDA to further understand the deficiencies and try to find a path forward, the company’s CEO Russel Trenary said in a statement. During a Wednesday call with analysts, Trenary and his team didn’t offer many details about the CRL. The company will have more to share about the agency’s concerns—and a timeline for resolution—after the FDA meeting, the CEO said. The CMC issues are “addressable” and “manageable,” he added. Outlook has hired Fujifilm Diosynth Biotechnologies and Ajinomoto Biopharma Services to help make its bevacizumab reformulation. Neither company responded Fierce Pharma’s request for comment by publication time. Avastin is approved by the FDA to treat various cancers thanks to its ability to prevent the growth of new blood vessels that feed tumors. This anti-VEGF mechanism is also well-accepted in the treatment of diseases featuring abnormal blood vessels in the back of the eye. Regeneron’s Eylea, plus Roche and Novartis’ Lucentis, are blockbuster VEGF inhibitors for the eye. Even without the FDA’s blessing, doctors have been using unapproved and repackaged bevacizumab provided by compounding pharmacies. These versions “have known risks of contamination and inconsistent potency and availability,” Outlook noted. Meanwhile, several Avastin biosimilars are available in the U.S. But these copycats can only officially go after the same indications as the originator. Twelve years ago, a collaboration between the University of Pennsylvania and the National Eye Institute found that Avastin and Lucentis had equivalent effects on improving visual acuity in wet AMD. But those results were never used for an FDA application. In Outlook’s phase 3 NORSE TWO trial, 42% of wet AMD patients who took Lytenava, also coded as ONS-5010, gained at least 15 letters (three lines) of vision in a common test. The trial met its primary and secondary endpoints with high statistical significance, Trenary stressed. Outlook has retained several former FDA reviewers and attorneys to help the company understand the FDA’s questions and to vet its answers, Trenary added. Despite the setback, the company is still targeting an FDA approval.