Axcella Health Inc. (AXLA)
First Day Turnover
We are a biotechnology company pioneering the research and development of novel multifactorial interventions to support health and address dysregulated metabolism across a broad spectrum of consumers and patients who have limited options. Our AXA Candidates are generated from our proprietary, human-focused AXA Development Platform and harness the power of endogenous metabolic modulators, or EMMs, a broad family of molecules that fundamentally impact and regulate human metabolism.
Using our AXA Development Platform, we have rapidly generated a pipeline of AXA Candidates that are novel compositions of EMMs engineered in distinct ratios and designed to target and maximize the fundamental role that EMMs play in regulating multiple metabolic functions. Our AXA Candidates are administered orally and anchored by amino acids, which have a history of safe use as food.
As such, we expect our AXA Candidates may also be combinable with other modalities. We believe our current dataset supports the potential of our AXA Candidates to support and modulate the metabolic pathways they target with favorable safety profiles. These features may make them an attractive development opportunity with significant commercial potential.
In 2018, we completed three Non-IND, IRB-Approved Clinical Studies. In all three studies, our AXA Candidates were generally found to be well tolerated, and we generated positive normal structure and function data indicating potential metabolic modulation. We believe generating this rich human dataset at this early stage of development (i) eliminates the translational uncertainty associated with transitioning from animal studies to human studies (ii) enables us to make high-insight, capital-efficient development decisions and (iii) potentially increases the probability of success for these AXA Candidates.
For AXA Candidates we decide to develop under an Investigational New Drug application, or IND, we will discuss with the U.S. Food and Drug Administration, or FDA our ability to use data generated from our Non-IND, IRB-Approved Clinical Studies to begin Phase II or potential registrational (pivotal) Clinical Trials.
On March 6, 2019, we had a face-to-face pre-IND meeting with the FDA for AXA1665, our lead AXA Candidate, during which we discussed clinical endpoints, assessment tools and other matters relating to a potential IND-opening Clinical Trial for AXA1665 in hepatic encephalopathy, or HE. After meeting with the FDA, we made a decision to pursue a drug development path for AXA1665. Under the planned IND, the initial indication would be for the treatment of HE, with time to breakthrough episode of overt HE event as the primary endpoint and key secondary muscle-related endpoints. We anticipate interacting with the FDA again prior to a formal IND submission for AXA1665. We anticipate initiating a Clinical Trial in the second half of 2020 for AXA1665 that we believe could potentially serve as a registrational (pivotal) trial to support the submission of a New Drug Application, or NDA.