Axcella Health, a biotechnology company pioneering the research and development of novel multifactorial interventions to address dysregulated metabolism and support health, announced key milestones in the development of AXA4010, the company’s first investigational hematology product candidate.
Mechanistic data on AXA4010 will be presented at the American Society of Hematology’s (ASH) 2019 Annual Meeting. Additionally, the company has initiated enrollment of subjects with sickle cell disease (SCD) in a non-IND clinical study.
AXA4010 is composed of a novel combination of endogenous metabolic modulators (EMMs) designed to affect multiple pathways related to red cell membrane biology, hemolysis, endothelial cell and vascular function, and inflammation. Subjects with SCD have multiple metabolic derangements, making this an appropriate population to evaluate the potential impact of AXA4010 on blood structure and function.
“AXA4010 represents Axcella’s expansion beyond liver and muscle programs and demonstrates the strength and breadth of our development platform,” said Bill Hinshaw, president and chief executive officer of Axcella.
“We are pleased that our data have been accepted for a presentation at ASH 2019, the world’s premier hematology congress. With a first clinical study now underway, we look forward to better understanding the safety and tolerability profile of AXA4010, investigating how it might influence blood physiology and, consequently, informing our overall development plans.”
Axcella will present mechanistic data related to AXA4010 at the ASH 2019 Annual Meeting being held December 7-10, 2019 at the Orange County Convention Center in Orlando, FL. The poster presentation will discuss metabolic profiling of SCD plasma samples and cellular data regarding the impact of AXA4010 on preclinical models of vascular adhesion, inflammation and red blood cell function.
Axcella has enrolled its first subject with SCD in AXA4010-001 (NCT04134299), which is being conducted at multiple U.S. sites. This study is expected to enroll approximately 24 subjects ages 12 and older in a staged sequential design of three separate cohorts for up to 12 weeks.
In addition to safety and tolerability, the study will assess the effects of AXA4010 on blood structure and function, including hemolysis, inflammation and vascular physiology. Axcella currently anticipates a data readout from this study in the second half of 2020.
Endogenous metabolic modulators (EMMs) are a broad family of molecules, including amino acids, which fundamentally impact and regulate human metabolism. Axcella’s AXA Candidates are comprised of EMMs that individually have a history of safe use as food. The company believes that, unlike conventional targeted interventions currently used to address dysregulated metabolism, EMM compositions have the potential to directly and simultaneously modulate multiple metabolic pathways implicated both in complex diseases and overall health.
Axcella conducts Institutional Review Board (IRB)-approved, non-investigational new drug application (Non-IND) clinical studies in humans with its AXA candidates under U.S. Food and Drug Administration regulations and guidance supporting research with food. In these studies, Axcella evaluates in humans, including in individuals with disease, AXA Candidates for safety, tolerability and effects on the normal structures and functions of the body.
Non-IND, IRB-Approved Clinical Studies are not designed or intended to evaluate an AXA Candidate’s ability to diagnose, cure, mitigate, treat or prevent a disease. If Axcella decides to further develop an AXA Candidate as a potential therapeutic, subsequent studies will be conducted under an IND.
Source: Company Press Release