SkQ1 Demonstrates Positive Topline Results in U.S. Phase 3 VISTA-2 Trial for Dry Eye Disease

On February 24, ESSEX Bio-Technology Limited and Mitotech jointly announced the positive top-line data from the VISTA-2 study—a randomized, double-blind, placebo-controlled, multicenter Phase 3 clinical trial of SkQ1 for the treatment of dry eye disease (DED) conducted in the United States. According to the press release, Mitotech will engage with the U.S. FDA this spring to determine the next steps in its development plan, with the goal of submitting a New Drug Application (NDA) in 2022–2023.

Publicly available information indicates that SkQ1 acts on mitochondria at the cellular level through an innovative mechanism to treat dry eye disease. Unlike most current treatments that primarily rely on anti-inflammatory mechanisms, SkQ1 targets and blocks ocular oxidative stress, thereby not only alleviating ocular inflammation but also improving issues such as degeneration of ocular surface tissues and reduced tear quality.

Dry eye disease is a chronic ophthalmic condition characterized by ocular discomfort and inflammation resulting from damage to the tear film. This damage may be caused by reduced tear production, an imbalance in tear composition, or excessive tear evaporation. The ocular discomfort and pain associated with dry eye disease can significantly impair patients’ quality of life, causing difficulties in reading, driving, using computers, or performing other daily activities. Currently, the primary conventional treatment for these patients is the use of artificial tears, which fails to adequately meet clinical needs.

VISTA-2 is a randomized, double-blind, placebo-controlled, multicenter clinical trial comprising two treatment groups receiving SkQ1 eye drops and placebo, respectively, twice daily. A total of 610 subjects participated in the U.S. clinical study and underwent a two-month treatment period.

One of the prespecified key secondary endpoints in the study (change from baseline in central fluorescein staining after only 28 days of treatment) showed a statistically significant improvement with SkQ1 treatment compared to placebo in the subgroup of subjects with higher tear secretion test scores (p<0.05). Notably, improvement in this endpoint was observed in this subgroup in both the VISTA-1 and VISTA-2 studies, resulting in a combined p-value of <0.0005 for the pooled data from the two studies. Furthermore, in both studies, the same subgroup of subjects demonstrated statistically significant clearance of central fluorescein staining (i.e., zero central corneal staining) and improvement in best-corrected visual acuity (BCVA) on Day 28 (p<0.05 for both endpoints in VISTA-1 and VISTA-2).

According to the press release, the data results from the above secondary endpoints provided clear guidance for setting the primary endpoint of Mitotech’s next pivotal clinical study aimed at a New Drug Application (NDA), thereby replacing the VISTA-2 trial’s composite clinical endpoint (conjunctival fluorescein staining and ocular discomfort on Day 56). The VISTA-2 study demonstrated that SkQ1 had a tolerability profile similar to that of artificial tears, further highlighting the drug’s excellent safety profile.

Mr. Yan Xianlong, Deputy Managing Director of ESSEX Bio-Technology Limited, stated: “The efficacy of SkQ1 in patients with dry eye disease (demonstrating excellent safety and tolerability) in the VISTA-1 and VISTA-2 trials has fully showcased its mechanism of action involving the inhibition of mitochondrial oxidative stress. We are excited by the data on the clearance of central corneal staining, which reveals the potential of SkQ1 in addressing oxidative stress in dry eye disease.”

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