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Acasti notes unusual placebo response in failed phase 3 heart drug trial

Acasti Pharma Inc.'s cardiovascular drug CaPre did not reduce triglycerides in patients with a chronic metabolic condition that heightens the risk of pancreatic and heart diseases, failing a phase 3 clinical trial.

CaPre was tested in patients with hypertriglyceridemia, which is characterized by high levels of triglycerides, a fat found in the blood. Patients with the condition have an elevated risk of developing pancreatic and heart diseases.

In the phase 3 trial called Trilogy 1, CaPre reduced triglyceride levels by 30.5% on average, compared to a 27.5% reduction seen in patients taking placebo at 12 weeks. Patients who received CaPre in addition to standard cholesterol therapy experienced an average triglyceride reduction of 42.2%, compared to a 31.5% reduction in patients in the placebo group. Acasti also reported a 36.7% average reduction in triglyceride levels for patients in the CaPre group at 26 weeks, compared to a 28% reduction in the placebo arm.

The Laval, Quebec-based pharmaceutical company said that Trilogy 1 did not achieve statistical significance due to an unexpectedly large percentage of patients who responded in the placebo group. CaPre's safety profile was similar to placebo as well. The company expects to report detailed results of the study by the end of the first quarter.

Simple cornstarch was used as a placebo in the study, surprising the company with a high response rate. Previous studies using corn oil, olive oil and vegetable oil as placebo experienced a maximum of 16% response in patients. Acasti is investigating the root cause of the significant placebo response as cornstarch is not likely the reason behind it.

Acasti said results from the ongoing Trilogy 2 study, which are expected in mid-February, may also provide useful data and a possible path forward to file an application with regulatory bodies for CaPre's approval.

Shares of Acasti plummeted as the U.S. markets opened Jan. 13, dropping more than 68% to 69 cents as of 11:25 a.m. ET. The shares previously closed at $2.25.