Esperion Therapeutics Inc announced the initial results from a Phase I clinical trial for a synthetic HDL comprised of ApoA-I Milano and phospholipid (AIM), ETC-216. With positive results on the safety and tolerability of AIM in healthy individuals after a single dose treatment, Esperion intends to initiate a clinical trial in patients later this year.

The Phase I trial was designed as a double-blind placebo-controlled study of 32 healthy volunteers, ages 18-50 years, to determine the safety and tolerability of five escalating doses of a single intravenous infusion of AIM in healthy males, and at two different dose levels in females. Volunteers were monitored for 27 days following dosing. The results indicate that AIM was safe at all doses, there were no serious adverse events observed, and AIM was well tolerated in the anticipated clinical dose ranges.

"We are pleased with the results we have seen thus far from the Phase I clinical trial," stated Roger Newton, president and CEO of Esperion Therapeutics. "We are now moving forward with our plans to initiate a clinical trial in patients with atherosclerosis. We are especially pleased that there was evidence of rapid mobilization of cholesterol consistent with preclinical studies to date."

AIM is a variant of ApoA-I, the major protein component of HDL. HDL is believed to protect against cardiovascular disease by efficiently removing cholesterol and other lipids from tissues including the arterial wall and transporting them to the liver for elimination. The clinical use of AIM as a human recombinant protein complexed to phospholipid is to mimic HDL and its function in a process known as reverse lipid transport (RLT).

AIM is present in a small population of Northern Italians with paradoxically low levels of HDL-cholesterol. Low HDL-cholesterol levels normally would correlate with high risk for cardiovascular disease, but carriers of the AIM gene show a reduced risk, presumably due to enhanced RLT. The clinical testing of AIM is a first for a human recombinant variant protein as a possible therapeutic agent to treat cardiovascular disease and its complications.