Axcella Presents Data at ASH Annual Meeting

Axcella Health Inc. (Nasdaq: AXLA), a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, today announced mechanistic data for AXA4010 that are being presented today at the American Society of Hematology’s (ASH) 2019 Annual Meeting.

AXA4010, Axcella’s first investigational hematology product candidate, is a novel EMM composition designed to target and impact multiple pathways related to red blood cell (RBC) membrane biology, hemolysis, endothelial cell and vascular function, and inflammation. The data being presented at the ASH Annual Meeting are from preclinical studies of constituents of AXA4010.

“Sickle cell disease (SCD) is a chronic hemolytic anemia that is associated with inflammation and metabolic derangements that include nitric oxide depletion and oxidative stress,” explains Dr. Shreeram Aradhye, M.D., Axcella’s Executive Vice President and Chief Development Officer. “As a result, SCD is an ideal setting in which to study AXA4010’s potential impact on multiple aspects of blood health. We are looking forward to leveraging these mechanistic data and findings from our ongoing non-IND clinical study of AXA4010 in subjects with SCD to inform our development plans for this candidate.”

Poster 978 presents the findings of the preclinical study, the highlights of which include:

• Plasma amino profiles of adults with SCD were significantly different from adults in the control group, with a 50 percent reduction in Arginine and more than a 30 percent reduction in total essential amino acids, which include Leucine, Valine, Lysine, and Histidine.

• Endothelial cell cultures treated with TNFα and constituents of AXA4010 saw reduction in markers of vascular adhesion, inflammation and cell migration. This was compared to cell cultures that were treated with glutamine and TNFα, which saw an increase in adhesion, no change in inflammation and a reduction in cell migration.

• Compared against individual amino acids, AXA4010 constituents were better at improving overall RBC deformability by improving membrane flexibility, surface-to-volume ratio and intracellular viscosity.

“These data again demonstrate the potential of EMMs to impact multiple dysregulated metabolic pathways,” said Bill Hinshaw, president and CEO of Axcella. “We are excited about the continued expansion of Axcella’s portfolio beyond our liver- and muscle-related programs, into hematology.”