PharmaMar suffers late-stage failure for RNAi-based therapy in dry-eye disease

2024-02-09

临床3期寡核苷酸siRNA

PharmaMar said Friday that a Phase III study of the RNAi-based therapy tivanisiran for the treatment of dry-eye disease associated with Sjögren’s Syndrome failed to meet its primary endpoint. Tivanisiran is being developed by the company’s Sylentis subsidiary and is its lead programme

The study randomised 203 patients dry-eye associated with Sjögren’s Syndrome to receive tivanisiran eye drops or vehicle once daily for 3 months. According to an entry on ClinicalTrials.gov, the main goals measured change from baseline in corneal fluorescein staining (CFS) scale and change from baseline in dry eye symptom scale.

Tivanisiran, also known as SYL1001, is a small synthetic double-stranded RNA oligonucleotide designed to selectively inhibit production of the transient receptor potential cation channel TRPV1.

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