Axcella Achieves Initial Milestones with its Hematology Product Candidate AXA4010
- Mechanistic data to be presented at ASH 2019 Annual Meeting
- Non-IND clinical study initiated to assess safety, tolerability and blood physiology; first subjects with sickle cell disease enrolled
- Axcella’s clinical pipeline expands beyond liver and muscle into hematology
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
ASH 2019 Presentation
Axcella will present mechanistic data related to AXA4010 at the ASH 2019 Annual Meeting being held
Abstract Number: #124224
Title: A Novel Composition of Endogenous Metabolic Modulators, AXA4010, Impacts Adhesion, Inflammation and RBC Membrane Deformability in Preclinical Models of Sickle Cell Disease
Presentation Type: Poster
Initiation of Non-IND Clinical Study
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.
About Endogenous Metabolic Modulators
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.
About Non-IND Clinical Studies
Internet Posting of Information
Axcella uses its website, www.axcellahealth.com, as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Such disclosures will be included in the “Investors and News” section of the company’s website. Accordingly, investors should monitor this portion of the website, in addition to following Axcella’s press releases,
Axcella is designing and developing AXA Candidates, compositions of endogenous metabolic modulators, or EMMs, engineered in distinct ratios, designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Axcella’s AXA Candidates are generated from its proprietary, human-focused AXA Development Platform. Axcella believes its expertise and capabilities in EMMs position it to become a preeminent biotechnology company reprogramming metabolism to address a diverse set of complex diseases and support health. Axcella’s AXA Development Platform has already produced a pipeline of product candidates in programs targeting liver, muscle and blood. Axcella was founded by Flagship Pioneering. For more information, visit www.axcellahealth.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the development potential of AXA Candidates, including AXA4010, potential expansion into new therapeutic fields, the ability of endogenous metabolic modulators to impact dysregulated metabolism and health and the timing of the company’s clinical studies and the timing of receipt of data from the same. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those related to the breadth of the company’s pipeline of product candidates, the strength of the AXA Development Platform, the efficiency of the company’s discovery and development approach, the clinical development and safety profile of AXA Candidates and their health or therapeutic potential, whether and when, if at all, AXA Candidates will receive approval from the