ANN ARBOR — Ann Arbor-based AlphaCore Pharma and Rockville, Md.-based Advanced Bioscience Laboratories Tuesday announced funding from the National Heart, Lung and Blood Institute, part of the National Institutes of Health, to manufacture a new treatment for a type of coronary artery disease that runs in families.

The funding comes under the NHLBI’s “Science Moving towards Research Translation and Therapy” (SMARTT) program. It will fund manufacturing of an enzyme called recombinant human lecithin-cholesterol acyltransferase (rhLCAT) for the treatment of a disease called familial LCAT deficiency.

Also known as ACP-501, rhLCAT represents a promising new approach in the fight against atherosclerotic cardiovascular disease, and has demonstrated preclinical efficacy in promoting HDL maturation and cholesterol flux, a natural process by which cholesterol is removed from the body.

Currently, ACP-501 is in Phase 1 clinical development with the eventual goal of reducing the risk of cardiovascular events in patients with acute coronary syndrome.

Manufacturing support from the NHLBI SMARTT program will enable production of additional material that will be used to determine the safety and efficacy of rhLCAT enzyme replacement therapy for patients with familial LCAT deficiency — a potentially life-threatening illness for which there is no FDA-approved treatment.

“This is a significant step towards developing a treatment for familial LCAT deficiency,” said AlphaCore president Bruce Auerbach. “We are pleased by the strong support from the NHLBI and ABL and look forward to advancing this program.”

The enzyme, rhLCAT, will be produced by ABL in its Rockville, Md. biologics production plant under a contract from the NHLBI SMARTT program.

“ABL is privileged to be working with AlphaCore Pharma in support of their ACP-501 program,” said Thomas VanCott, ABL president and CEO. “Research in rare genetic diseases can encounter funding hurdles, yet through this NHLBI-sponsored manufacturing project we have the potential to advance an urgently needed enzyme replacement therapy. This effort further demonstrates ABL’s expertise of partnering with the NIH to support major development programs and our commitment to deliver the highest quality cGMP biologics to our clients in a cost-effective manner.”

AlphaCore Pharma is a private company focused on the development of ACP-501 (recombinant human lecithin: cholesterol acyltransferase or rhLCAT) as a therapy for acute coronary syndromes, and other high-risk atherosclerosis conditions. Additionally, ACP-501 is being developed to treat familial LCAT deficiency (an orphan designated enzyme replacement therapy) and other diseases where LCAT activity is low. Additional information on AlphaCore Pharma is available at


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