Otsuka Pharmaceutical Co., Ltd (Otsuka) announces that the primary endpoint was achieved in the Phase 3 trial in Japan of bempedoic acid (chemical name) as a potential treatment for patients with hypercholesterolemia, The trial demonstrated statistically siginficant outcomes and highlighted its potential future value of bempedoic acid to patients in Japan.

Bempedoic acid, created by Esperion Therapeutics, Inc. (Esperion), has a novel mechanism of action that inhibits a cholesterol synthesis pathway by acting on ATP (adenosine triphosphate) citrate lyase, a citrate-degrading enzume in the liver. Bempedoic acid is marketed for the treatment of hypercholesterolemia in several regions around the world, including the United States and Europe.

In 2020, Otsuka acquired exclusive development and commercialization rights for bempedoic acid in Japan from Esperion and is currently developing it domestically.

(https://www.otsuka.co.jp/en/company/newsreleases/2020/20200420_1.html)

This Phase 3 trial (NCT05683340) was conducted as a placebo-controlled, randomized, multicenter, double-blind, parallel-group comparative study, in 96 patients with high LDL cholesterol and in whom statins have insufficient effect or cannot be tolerated. Trial participants were administered either 180 mg of bempedoic acid or a placebo, orally, once a day, for 12 weeks to evaluate the efficacy and safety of bempedoic acid.

In the preliminary results, the percentage change from baseline in LDL-C at Week 12, the primary endpoint, was -25.25% in the group receiving bempedoic acid group and -3.46% in the placebo group, demonstrating positive outcomes with statistical significance compared to placebo (p<0.001). Furthermore, the safety and tolerability of bempedoic acid were consistent with findings from previous trials, and no serious adverse events were observed.

Further data analysis will be conducted, and Otsuka plans to announce the results at a professional conference. Based on the results of this trial, Otsuka plans to submit an NDA (New Drug Application)　 in Japan in the latter half of 2024.