Axcella touts interim NASH data but needs the cash to stay afloat for 1H24 topline

Axcella Therapeutics, with an empty bank account looming in the first quarter of next year and a recent setback in a mid-stage long Covid study, is out with some interim data on its NASH drug.

Margaret Koziel

The biotech isn’t yet saying how the drug performed on the primary endpoint of improvement in the nonalcoholic fatty liver disease (NAFLD) Activity Score (NAS), but the first batch of results has sent shares $AXLA soaring nearly 15% before Thursday’s opening bell.

At 24 weeks, Axcella said all patients taking the high dose of its drug, AXA1125, showed statistically significant improvement in liver stiffness measurement compared to placebo, coming in at a p-value of 0.0096 with an absolute change in LSM at -4.07 kilopascals, or kPa. The low-dose arm did not reach statistical significance with -2.01 kPa and a p-value of 0.0992. Positive changes in liver stiffness are correlated with improvement in fibrosis, CMO Margaret Koziel said in a statement.

An Axcella spokesperson told Endpoints News via email that the company is “evaluating various options including financing and business development opportunities to fund the organization.”

Axcella is trying to position the oral drug — a combination of six amino acids and derivatives — as a treatment for NASH and long Covid, with the latter not meeting the bar in a Phase II primary endpoint, but with the biotech nonetheless attempting to move forward on fatigue measures.

“[The results] indicate 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,” CEO Bill Hinshaw said in a statement Thursday.

The preplanned interim analysis, conducted once 30% of 270 expected patients were enrolled, looked at 82 patients at week 12 and 58 who had thus far made it 24 weeks. The ongoing blinded study randomized patients 1:1:1 to either placebo, 22.6 g or 33.9 g of AXA1125 twice daily.

At both time points, all patients analyzed were shown to have statistically significant improvements in alanine aminotransferase (ALT), which is indicative of liver cell inflammation, Axcella said. The improvements came in at -28.61% (p=0.0183) for the low dose and -36.3% (p=0.0017) for high dose.

A field littered with clinical trial failures, NASH has long been a snag for drug developers, but a recent crop of mid-stage data out of Akero Therapeutics sent the biotech straight to a $175 million public offering after the company said its drug led to at least one stage of improvement in fibrosis with NASH worsening in 41% of patients on 50 mg and 39% of those in the 28 mg arm.

Axcella will soon need to raise money if it wants to make it to the topline 48-week biopsy results for AXA1125. That Phase IIb data readout is on tap for the first half of 2024, Hinshaw said, but the company only has the capital to keep the lights on into the first quarter of 2023, Axcella reported in earnings last month.

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