Despite its relative youth, the company has a rich pipeline as of June 2018 with candidates in the muscular, nervous and mitochondria spaces among others.

The company attracted attention in mid-2018 for attracting a Novartis executive, William Hinshaw, to leave big pharma for the promising small biotech.

In September 2022, Axcella said its experimental treatment for the challenging disease area of non-alcoholic steatohepatitis (NASH) showed statistically-significant improvement in liver stiffness.

The interim analysis of a Phase IIb indicates that administration of AXA1125 over 24 weeks leads to statistically-significant improvements compared to placebo in biomarkers for metabolism, inflammation and fibrosis, underscoring its multi-targeted efficacy.

Top-line, 48-week biopsy results are expected to be reported in the first half of 2024.