Our lead product, SPVN06 is a breakthrough therapy for retinitis pigmentosa (RP), an orphan inherited retinal disease which leads to blindness and affects nearly two million individuals worldwide. SPVN06 uses a gene therapy-based approach agnostic of mutated genes. It express proprietary neurotrophic factors and is delivered via a single subretinal injection. SPVN06 aims to prevent the degeneration of photoreceptors leading to blindness.
Unlike traditional gene therapies which replace a specific defective or missing gene in a given cell, SPVN06, our lead drug product, acts independently of the causative gene mutation. SPVN06 introduces RdCVF and RdCVFL, two neurotrophic factors naturally produced by rods and cones in normal conditions, in patients experiencing a significant deterioration of the photoreceptors (rods and cones). The DNA of the two distinct isoforms (RdCVF and RdCVFL) of the NXNL1 gene are supplied via an associated-adenovirus (AAV), a viral vector classically used in gene therapy. SPVN06 is delivered via the subretinal route to the cones and retinal pigmented epithelium (RPE). Such route of administration is already used in ophthalmology, for instance with Luxturna [Russel et al;, 2017). Upon delivery of SPVN06, the transgenes are mainly expressed in the RPE cells for RdCVF and in the cones for RdCVFL. SPVN06 is expected to provide a long-lasting neuroprotective effect to prevent the progression of RP in patients with moderate to severe pathology.
As of October 2020, the pharmaceutical development of SPVN06 is well advanced, with successful manufacturing of the pilot runs, and a first GMP production lot ongoing. The first GMP production will provide SparingVision with the clinical material needed for the Phase 1/2 clinical trials. In parallel, IND-enabling studies are ongoing to assess SPVN06 pharmacology, biodistribution and safety in rodent and non-rodent species.
The drug product and this mutation-agnostic approach are protected by several patent families.
SPVN06 has been granted Orphan Drug Designation in Europe and interactions with the US and EU regulators have been initiated in view of a CTA and IND filing in the second half of 2021.
The Company plans to start its first in human trial in the second part of 2021. The first clinical trial will be a Phase I/II dose escalating study aimed at testing the safety and tolerability of SPVN06 on a limited number of patients. The study will then be extended to test the efficacy of the recommended Phase 2 dose. Initial safety data are expected in the second half of 2022. The second clinical study will be initiated thereafter once longer term safety and efficacy data has been obtained in a larger cohort of patients.
SparingVision’s goal is to bring SPVN06 as rapidly as possible to the millions of patients suffering across the globe from this irremediable disease which causes visual dysfunction and degeneration and leads to blindness.
Russel et al., 2017. https://pubmed.ncbi.nlm.nih.gov/28712537/