MedImmune makes a move

GAITHERSBURG, Md.—In a move that gains it a new potentialdrug candidate in cardiovascular health, MedImmune, the global biologicsresearch and development arm of AstraZeneca, has acquired Ann Arbor,Mich.-based AlphaCore Pharma. AlphaCore is a biotechnology company focusing ondeveloping its lead compound, ACP-501, a recombinant human lecithin-cholesterolacyltransferase (LCAT) enzyme.

No financial details for the transaction were disclosed.

Dr. Cristina Rondinone, vice president and head of theCardiovascular/Metabolic Disease Innovative Medicines unit at MedImmune, saysthat the novelty of the company made it attractive as an acquisition target.She notes that MedImmune believes the LCAT enzyme is "key in cholesteroltransport," and says the company is optimistic about ACP-501 given its safetyprofile.

"We are very excited about the novelty of this drug,"Rondinone says. "In everything that we do in biologics … our goal is not onlyto treat, but to reverse diseases. We believe that with this drug, we can make adifference, and we are extremely excited about that."

LCAT is found in the bloodstream, and exists as a key factorin the reverse cholesterol transport system, which is believed to havesignificant importance in driving the removal of cholesterol from the body. Itis also thought to be pivotal in managing high-density lipoprotein (HDL), or"good" cholesterol, levels. The LCAT enzyme is also thought to factor into arare hereditary disorder known as familial LCAT deficiency, in which thisenzyme is missing.

"As the science in this area continues to evolve, we arecommitted to exploring unique pathways that could lead to new combination orstandalone therapies for patients living with chronic and acute cardiovasculardiseases," Dr. Bahija Jallal, executive vice president of MedImmune, said in apress release. "Cardiovascular disease is projected to remain the singleleading cause of death worldwide over the next decade and beyond. Through novelapproaches like LCAT, we hope to shift the treatment paradigms in this area tohelp prevent and treat these conditions."

AlphaCore is developing its compound as a novel therapeuticagent for treating high-risk atherosclerosis and serious lipid metabolismdisorders, and ACP-501 was granted orphan drug designation from the EuropeanMedicines Agency for the treatment of familial LCAT deficiency in November2012.

In October 2012, the company released results from its PhaseI clinical trial of the drug, announcing that ACP-501 had met its primaryendpoint by demonstrating its safety and tolerability, as well as its secondaryendpoints by rapidly and substantially elevating HDL cholesterol. The trial wasan ascending dose study of 16 patients with stable atherosclerosis, and noserious adverse events were reported.

"Weare gratified that ACP-501 was shown to be safe in this trial, and moreover, thatthe changes in biomarkers are consistent with rapid enhancement of reversecholesterol transport," Bruce Auerbach, president of AlphaCore, commented in anannouncement regarding the trial results. "The increase in HDL cholesterolafter administration of ACP-501 supports the notion that LCAT is a bottleneckin the reverse cholesterol transport process."

Cardiovascular and cardiometabolic disease are areas of keytherapeutic interest for AstraZeneca, and MedImmune boasts a cardiovascularpipeline as well. It is a market that Rondinone calls huge. It is not simply amatter of cardiovascular patients, she notes, but also a matter of the obesityepidemic and rising numbers of diabetes patients, which have led to increasedcardiovascular risk. Indeed, heart disease remains the leading cause of deathin the United States, according to the Centers for Disease Control andPrevention, with nearly 600,000 deaths recorded in 2010. The World HealthOrganization rated ischemic heart disease as the leading cause of deathworldwide as well for 2008, with a recorded 7.25 million fatalities,representing a full 12.8 percent of deaths for that year.