Safety profile for sepofarsen for Leber congenital amaurosis ‘manageable’
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Twelve-month results from a phase 1/2 trial of sepofarsen for Leber congenital amaurosis type 10 showed a manageable safety profile in adults and children, according to a poster presented at the virtual American Academy of Ophthalmology meeting.
Intravitreal sepofarsen (ProQR Therapeutics) is under study “as a potential injectable therapeutic anti-sense messenger RNA sequence that fixes the LCA10 host messenger RNA splicing defect that leads to LCA10 blindness. Preliminary results demonstrate a manageable safety profile and signs of potential vision improvement,” co-author Allen C. Ho, MD, told Healio/OSN.
“Progress for retinal degenerations continues and as the public becomes more aware of gene-based therapies or messenger RNA-based therapies (think recent Pfizer and Moderna COVID vaccine preliminary data) for a variety of human afflictions, the public should be aware that DNA and RNA strategies are being employed for causes of blindness,” Ho said.
Primary outcome of the first-in-human, open-label, multidose, dose-escalation trial in 11 subjects was safety and tolerability, with cataracts identified as the primary adverse event with earlier onset observed in the higher dose cohort.
Standard cataract surgery in six subjects returned vision to pre-cataract acuity. Cystoid macular edema and retinal thinning were observed only in the higher dose cohort.
All injection related adverse events were self-limited, and there were no systemic adverse events.
“I am cautiously optimistic about the possibilities of this specific program and the potential of the phase 3 Illuminate clinical trial as well as the opportunities for anti-sense RNA sequence strategies as a platform for therapy of other diseases,” Ho said.
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Russel SR, et al. Phase 1b/2 trial results of intravitreal sepofarsen RNA therapy in leber congenital amaurosis 10. Presented at: American Academy of Ophthalmology annual meeting; Nov. 13-15, 2020 (virtual meeting).
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