Retinitis Pigmentosa Gene Therapy Cleared for Trials
Initial safety data from the PRODYGY study are expected in 2023.
The FDA has cleared the investigational new drug application (IND) of SparingVision’s gene therapy SPVN06 for treating retinitis pigmentosa (RP).
“RP is a highly prevalent eye disease leading inevitably to blindness with no treatment available for the vast majority of patients. The unique neuroprotective mechanism of action of SPVN06 has the potential to change the course of the natural history of the disease, independently of the genetic background of patients and of the time of disease diagnosis. With this Phase 1/2 trial we will be looking to demonstrate SPVN06’s safety and tolerability as well as identify preliminary signs of efficacy through several structural, functional and quality of life endpoints,” Daniel Chung, chief medical officer, SparingVision, said in a statement.
Following the IND clearance, SparingVision will initiate the first-in-human Promising Rod-cone Dystrophy Gene therapy (PRODYGY) phase 1/2 clinical trial shortly, with initial safety data expected in 2023 and the primary endpoint expected to be reached in 2025. PRODYGY will recruit up to 33 participants with RP due to a mutation in the RHO, PDE6A, or PDE6B gene to receive a single subretinal injection of SPVN06 in their worst-seeing eye.
The study will begin with an open-label, dose-escalation phase of 3 cohorts of 3 participants with advanced forms of RP to determine the phase 2 recommended dose for the second phase. Cohorts 2 and 3 will be enrolled based off the decision by a Data Safety Monitoring Board (DSMB) after reviewing all available safety data from the prior cohorts. The second, randomized, double-blind, controlled phase of the trial will enroll 24 participants with intermediate RP to be divided into 3 cohorts of 6 participants, with 6 patients in the untreated control group and 18 receiving a low or high dose of SPVN06.
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PRODYGY is primarily evaluating safety and tolerability of SPVN06 12 months after treatment. The study will also evaluate preliminary efficacy and quality of life data as secondary outcomes and will continue to assess safety and tolerability in long-term follow-up for 5 years after treatment. SparingVision’s clinical trial authorization (CTA) application in France is also currently under review.
SPVN06 is a gene therapy that aims to stop or slow disease progression of inherited retinal diseases and age-related macular degeneration and regardless of genetic background, with mid-stage RP as an initial focus. The therapy is designed to restore the neurotrophic factor RdCVF by delivering 2 distinct isoforms (RdCVF and RdCVFL) of the NXNL1 gene via an adeno-associated virus vector, administered by subretinal injection.
“Receiving IND clearance is a testament to the incredible efforts of everyone at SparingVision, but also to the strength of our science, which encompasses more than 20 years of leading research by our scientific founders, including Professor José Sahel, Chair of the Ophthalmology Department at the University of Pittsburgh Medical Center. With over 80 genes involved in RP, each with numerous causative mutations, we need to go beyond the gene-by-gene treatment approach. SPVN06 has the potential to become the universal therapeutic solution that patients need, and we are excited for the next phase of development,” Stéphane Boissel, president and chief executive officer, SparingVision, added to the statement.
