Galecto has linked its myelofibrosis candidate GB2064 to reduced collagen fibrosis in a small clinical trial, offering early validation of the ability of the LOXL2 inhibitor to act on a variable that affects outcomes.
The analysis covers five patients who received GB2064 as a monotherapy for at least six months in the MYLOX-1 trial. Four of the patients experienced a one grade or greater reduction in collagen fibrosis of the bone marrow. The severity of bone marrow fibrosis correlates with clinical manifestations and affects survival.
Galecto previously identified reduced fibrotic bulk and more blood formation space as potential benefits of GB2064. The interim analysis provides early evidence to support the theory, although it remains to be seen whether the drug candidate can make a meaningful difference to clinical outcomes.
All of the fibrosis responders had stable hematological parameters, namely hemoglobin, white blood cell count and thrombocytes, and stable spleen volume over the six-month treatment period. None of the four patients required transfusion. Two of the subjects have entered the extension phase of the trial. In a statement, Claire Harrison, the safety committee chair, said “stable disease is excellent” in myelofibrosis.
The interim analysis is a staging post on Galecto’s journey to full data from the study, which will track patients for nine months of treatment. Half of the 16 patients dosed so far have discontinued treatment as a result of an adverse event or disease progression. The most common adverse events were gastrointestinal and “manageable” in most patients with standard therapy, according to Galecto.
It is unclear exactly when Galecto will have more data from the trial. In a statement to disclose the data, the biotech said it will provide an update on the timing of the full readout later this year and continues to evaluate whether to make adjustments to facilitate patient recruitment.