Axcella Therapeutics has presented a glimpse at data from its ongoing, blinded phase 2b nonalcoholic steatohepatitis (NASH) trial, linking the high dose of its orally active mixture of amino acids to improved liver stiffness at week 24.
The EMMPACT study is primarily designed to test whether 48 weeks of twice-daily oral doses of AXA1125 can cause a biopsy-confirmed two-point improvement in non-alcoholic fatty liver disease activity scores. That question remains unanswered, for now, but the interim analysis triggered by the enrollment of 30% of the targeted 270 participants gave Axcella a chance to look at data on other endpoints.
Although the study remains blinded, the biotech reported that absolute changes in liver stiffnesses, as measured by vibration controlled transient elastography, at week 24 were 0.13 and -4.07 kilopascals in the placebo and high dose arms, respectively. The difference was statistically significant.
While the low dose liver stiffness result, -2.01, fell short of statistical significance, Axcella linked it to improved outcomes on other measures. Axcella reported statistically significant improvements in ALT, a liver enzyme, at week 12 and 24, and changes in liver fat, as measured by MRI, at week 12 at both doses. The liver fat result wasn’t significant at week 24.
In a release, Axcella CEO Bill Hinshaw called the results “extremely encouraging.” However, they may mean little if the study misses the primary and secondary endpoints that require the collection of biopsy data at week 48.
As it stands, there are questions about Axcella’s ability to get to those key readouts. The biotech expects to report the topline, 48-week biopsy results in the first half of 2024. Last month, Axcella told investors the $44.4 million it had in cash at the end of June should keep it going into the first quarter of 2023. If a loan repayment is sought, the biotech could reach the end of its cash runway before the end of 2022.