Kubota Pharmaceutical Holdings Co., Ltd.
Positive post-hoc analysis presented for Phase 3 clinical trial Emixustat hydrochloride, a drug candidate for the treatment of Stargardt’s disease
Kubota Pharmaceutical Holdings Co., Ltd. (Headquarters: Chiyoda-ku, Tokyo, hereinafter “the Company”) has announced that it will launch “Emixustat Hydrochloride” (hereinafter “Emixustat”) implemented by Kubota Vision Inc. (Headquarters: Washington, USA), a wholly owned subsidiary of the Company. ) for the indication of Stargardt’s disease.
The primary endpoint of this clinical trial includes verification of the efficacy in suppressing the progression of macular atrophy lesions in patients with juvenile macular degeneration Stargardt’s disease. There were no results demonstrating statistical significance for the emixustat-treated group compared with Subsequent further analysis suggested a significantly lower rate of progression of atrophic lesions in the emixustat group compared to placebo in the group of subjects with smaller baseline atrophic lesion area (mm2). , we conducted a subgroup analysis (*1) to verify this. We performed univariate and multivariate analyzes by attrition on the group of subjects with small baseline atrophic lesion area to identify independent baseline factors influencing the progression of atrophic lesions in this subgroup. The analysis showed that the progression rate of macular atrophy at 24 months in the emixustat group was 40.8% compared to the placebo group.
%reduced (p=0.0206, emixustat n=34, placebo n=21).
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 has been completed as scheduled.
Our Chairman, President and Chief Executive Officer, Dr. Ryo Kubota commented as follows.
“We are very pleased with the results of the subgroup analysis of this clinical trial, which showed high efficacy. It suggests the importance of early detection and early treatment as well as the fact that there are people with Stargardt's disease.
In the future, we will continue to search for joint development partners and other activities, and will reconsider Emixstat's future plans. At the same time, we will focus on the development and sales of medical equipment centered on Kubota eyeglasses.
We are currently investigating the impact of this matter on the consolidated performance for the fiscal year ending December 2022, and will promptly disclose any revisions to our performance forecasts if necessary.
*1 Subgroup analysis is the extraction and analysis of a specific group from the entire population.
About the grant program for this clinical trial
In August 2020, this clinical trial was selected by the FDA as a grant program for the Orphan Products Clinical Trials Grants Program. Kubota Vision invested 22.85 million dollars (approximately 3 billion yen) (93.3%), and received a total of 1.63 million dollars (approximately 220 million yen) (6.7%) from the U.S. Department of Health and Human Services (HHS), which has jurisdiction over the FDA. I was. Please note that the content of this press release has been issued by the Group and does not necessarily represent the official opinion or approval of the FDA/HHS or the US government.
About Stargardt Disease
Stargardt's disease is a genetic disease of the retina with a small number of patients. When Stargardt disease develops, photoreceptor cells are gradually damaged, and various symptoms such as loss of visual field, color blindness, distortion, blurring, and difficulty seeing in the center can be seen. Although it typically begins in childhood or adolescence, some people do not notice the loss of vision until adulthood. Stargardt's disease is estimated to affect less than 40,000 people in the United States (*3). 4)”. Currently, there is no therapeutic drug to suppress the progression of symptoms, and it is reported that the market for Stargardt's disease will reach
approximately 160 billion yen in 2027 (*5).
*2 Retinal Pharma & Biologics Market, Market Scope 2015.
*3 Based on )
https://www.kubotaholdings.co.jp)Disclaimer
This material has been prepared for the sole purpose of disclosing relevant information and does not constitute a solicitation or offer to acquire or sell securities. This document has been carefully prepared for accuracy, but completeness is not guaranteed. In preparing this material, we relied on third-party information available to us, but we do not represent or guarantee the actual accuracy or completeness of such third-party information. Our company does not take any responsibility for any damage caused by relying on the descriptions in this document. Information in this document is subject to change without notice. This document contains
forward-looking information regarding future forecasts. These statements are based on current expectations, forecasts and risky assumptions, including but not limited to general economic and industry or market conditions, and may differ materially from actual performance. There is a possibility. future, new information, future We undertake no obligation to update or revise any forward-looking statements in this material, whether for events or otherwise. Details about this release:
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