Kubota Vision Announces Positive Post Hoc Analysis from Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease
Kubota Vision announced positive results from a post hoc analysis of the phase 3 clinical trial of the investigational visual cycle modulator emixustat hydrochloride (emixustat) in patients with Stargardt Disease.
The primary objective of this study was to determine if emixustat reduces the rate of macular atrophy progression, in comparison to placebo, in subjects with Stargardt disease. Previously announced topline results showed that the study did not achieve its pre-planned primary or secondary endpoints. Prompted by planned subgroup analyses suggesting that emixustat subjects with smaller atrophic lesions at baseline had a reduced rate of progression compared to placebo subjects, post hoc analyses were performed to further investigate this finding. A multi-factor analysis was performed on the subgroup of subjects with smaller lesions at baseline, controlling for the baseline factors identified in univariate and multi-factor analyses to affect lesion progression in this subgroup. This analysis determined that emixustat treatment resulted in a 40.8% reduction in lesion progression compared to placebo at Month 24 (P=0.0206, emixustat n=34, placebo n=21).
The study was a multicenter, randomized, double-masked, and placebo-controlled phase 3 clinical study in which subjects were randomly assigned to emixustat 10 mg or placebo (2:1 ratio) once daily for 24 months. The total target number of subjects was 162; however, due to high interest in the study, a total of 194 subjects were enrolled in this study across 29 sites in 11 countries worldwide.
“We are very excited to see strong efficacy in this subgroup. The fact that we have shown a positive effect in an earlier stage of a disease is very similar to what has been observed in drug development for other neurodegenerative diseases such as Alzheimer’s disease. This offers the potential to treat these patients proactively with the goal to preserve vision. We will move forward quickly to develop emixustat in this population and will seek a partner for this development," Ryo Kubota, MD, PhD, Chairman, President, and CEO of Kubota Vision Inc., said in a company news release.
The company is examining the impact on the consolidated earnings forecast for the fiscal year ending December 31, 2022 (January 1, 2022 to December 31, 2022) and will be promptly disclosed if a revision to the financial forecast becomes necessary.
About Funding for this Clinical Trial
As previously announced in August 2020, the FDA Office of Orphan Products Development (OOPD) awarded Kubota Vision an orphan products clinical trial grant to support this clinical trial. Specifically, this clinical trial was supported by the FDA of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award (FAIN: R01FD006849) totaling $1,633,917 with 6.7% funded by FDA/HHS and $22,858,571 (93.3%) funded by Kubota Vision. The contents of this press release are those of Kubota Vision and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.