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GB2064 is an investigational, potential first-in-class, oral lysyl oxidase-like 2 (LOXL2) inhibitor intended for the treatment of patients diagnosed with myelofibrosis who are ineligible, intolerant, or refractory to Janus kinase inhibitor therapy. LOXL2 plays a key role in myelofibrosis, contributing to the fibrotic progression of disease via mediation of collagen crosslinking in tissue fibrosis. Recently, topline results from the phase IIa MYLOX-1 trial (NCT04679870), investigating the safety and efficacy of GB2064, were released. We summarize the key points below.
These topline results indicate that GB2064 elicits strong antifibrotic activity and inhibition of LOXL2 in a difficult-to-treat patient population. The safety profile was considered acceptable, and AEs were manageable. Further analysis is ongoing to determine the potential of subsequent clinical trial stages.
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