Tinlarebant shows promise in slowing Stargardt disease progression in adolescents
Tinlarebant, an oral retinol-binding protein 4 antagonist, appears to be a safe and effective treatment that may slow disease progression in adolescents with Stargardt disease by reducing retinol delivery to the eye, according to a poster presented at AAO 2024.
The 24-month open-label Phase 2 trial included 13 patients aged 12-18 years with biallelic ABCA4 variants and minimal autofluorescence changes at baseline. Patients received a daily dose of 5 mg tinlarebant over 24 months.
Treatment led to a sustained reduction in RBP4 levels, which was reversible after a 28-day cessation. No new definitely decreased autofluorescence lesions were observed in 5 patients with severe ABCA4 mutations. However, disease progression varied even among siblings with the same genetic mutations. Best corrected visual acuity remained stable, with an average loss of 5 letters over the study period. All adverse events reported were mild.
Reference
Mata NL, et al. Investigation of an Oral Retinol Binding Protein 4 Antagonist in the Treatment of Childhood-Onset Stargardt Disease. Poster presented at: American Academy of Ophthalmology Annual Meeting; October 2024; Chicago.
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