Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the Phase 3 ODYSSEY JAPAN trial of the investigational therapy Praluent® (alirocumab) Injection met its primary endpoint. At week 24, patients in the Praluent group experienced an average 64 percent greater reduction from baseline in their bad cholesterol, known as low-density lipoprotein cholesterol (LDL-C), when added to current standard of care including statins, compared to standard of care alone (p less than 0.0001). Patients were started on the lower dose of 75 mg, with the option to adjust their dose to 150 mg if they had not achieved their LDL-C goal (as defined by the Japan Atherosclerosis Society guidelines) at week 8. At week 24, 97 percent of patients in the Praluent group reached their LDL-C treatment goal, compared to 10 percent for placebo (p less than 0.0001). Ninety-nine percent of patients treated with Praluent remained on the lower dose; two patients required adjustment to the higher dose.
The trial involved 216 Japanese patients with hypercholesterolemia at high cardiovascular (CV) risk and/or with an inherited form of high cholesterol known as heterozygous familial hypercholesterolemia (HeFH). Results were presented today for the first time at the Annual Scientific Meeting of the Japan Atherosclerosis Society (JAS) in Sendai, Japan. Praluent is an investigational fully human monoclonal antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9).
"These results demonstrate the significant cholesterol-lowering ability of Praluent among patients with some of the greatest unmet needs in Japan. This includes those with an inherited form of high cholesterol or pre-existing cardiovascular disease, such as a history of heart attack," said lead investigator Tamio Teramoto, MD, PhD, Director of Teikyo Academic Research Center. "Despite current treatment options, many Japanese patients with hypercholesterolemia are still unable to reach their LDL-C goals, highlighting the need for additional treatment options. Notably, almost all patients reached their LDL-C target levels while remaining on the 75 mg dose, avoiding the need for overtreatment."
ODYSSEY JAPAN evaluated Praluent (n=144) compared to placebo (n=72), both on top of standard care, in Japanese patients with hypercholesterolemia, with either HeFH or at high CV risk, and who could not reach their LDL-C treatment goal as defined by the JAS guidelines despite lipid-lowering treatments that included statins. The mean LDL-C value at baseline was 141.2 mg/dL. Patients were initially randomized to receive either Praluent 75 mg every two weeks administered as a single 1 milliliter (mL) injection, or placebo. Patients in both groups received statins, with or without other lipid-lowering therapies.
Ninety-nine percent of patients who received Praluent at week 8 remained on the initial 75 mg dose, while one percent of patients had their dose adjusted to receive 150 mg every two weeks, also as a single 1 mL injection. The most common adverse events (occurring in at least 5 percent of patients in the Praluent group) were nasopharyngitis, injection site reaction, and back pain.
On June 9, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) recommended the approval of Praluent. The FDA has a target action date of July 24; and while the FDA takes the Committee's advice into consideration, the FDA is not bound by its recommendation. In addition, the Marketing Authorization Application for Praluent in the European Union is currently under review by the European Medicines Agency (EMA). The safety and efficacy of Praluent have not been fully evaluated by any regulatory authority.
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