Tivanisiran

February 9, 2024 The study that evaluated the efficacy of the drug did not meet the primary endpoint. Sylentis, a wholly owned subsidiary of PharmaMar (MSE:PHM), has obtained the results of the Phase III SYL10111_V (tivanisiran) clinical trial for the treatment of dry-eye associated with Sjögren’s Syndrome. The primary endpoint was to evaluate the efficacy (signs and symptoms) and was not met. The trial was a randomized, double-masked, placebo-controlled study conducted in more than 40 hospitals in the United States and 8 in Spain involving 203 patients. Sylentis would like to thank the patients, families, the hospitals, caregivers and the medical teams that have made it possible to carry out this trial.

FYDES achieved the primary endpoint of the study by demonstrating the long-term safety of tivanisiran ophthalmic solution administration. 301 patients with dry eye disease recruited from 26 centers in the United States participated. Sylentis, a wholly owned subsidiary of PharmaMar (MSE:PHM), has obtained the results of the FYDES clinical trial to evaluate the long-term safety of tivanisiran as eye drops for the treatment of adults with dry eye disease. This clinical trial was conducted at 26 centers in the United States and involved 301 patients. FYDES is a Phase III, randomized, placebo controlled, multicenter study, in which the safety of tivanisiran sodium ophthalmic solution was evaluated after being administered to patients with dry eye disease, once daily for approximately 360 days. The FYDES safety study is part of the tivanisiran development plan along with the SYL1001_V efficacy study in patients with dry eye disease associated with Sjögren's Syndrome and whose results will be available in the first quarter of 2024. Of the 301 patients randomized, 203 were assigned to the tivanisiran group and 98 to the control arm (2:1 ratio). Analysis of the primary endpoint showed that the overall frequency and percentage of patients experiencing at least one adverse event (ocular or non-ocular, related or unrelated) was similar in both treatment groups: 40.4% in the tivanisiran group and 39.8% in the control arm. The only adverse events related to tivanisiran were conjunctival hyperemia (1%), eye pruritus (1%), papillary conjunctivitis (0.5%) and blurred vision (0.5%). After one year of treatment, no clinically significant changes in other ocular safety parameters, laboratory values or body temperature have been observed. At upcoming medical meetings additional data and analyses will be submitted for presentation. Dry eye disease is a pathology that affects more than 300 million people worldwide and for which the available therapeutic options are currently very limited. This disease is one of the most frequent causes of consultation to the ophthalmologist and occurs when the eye does not produce tears correctly or when they do not have the necessary consistency and evaporate very quickly. It especially affects people in developed countries, where air pollution, air conditioning, the use of contact lenses, refractive surgery or the continuous use of computers are major risk factors. Some of the most notorious symptoms of the pathology are pain, burning, incessant itching, eye fatigue, dryness, blurred vision, sensitivity to light or foreign body sensation.