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Treatment sequencing for anemic myelofibrosis

with Jean-Jacques Kiladjian & Angela Fleischman

Monday, February 26, 2024 | 16:30 CET

This independent educational activity is supported by Bristol Myers Squibb. All content is developed independently by the faculty. The funder is allowed no influence on the content of this activity.


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MYLOX-1 trial: LOXL2 inhibition for the treatment of MF

Jan 22, 2024
Learning objective: After reading this article, learners will be able to cite a new clinical development in myelofibrosis.

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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.

Study design1

  • Phase IIa, open-label, single-arm study
  • 1,000 mg orally, twice daily for 9 months
  • Bone marrow biopsies at 3, 6, and 9 months
  • The primary study endpoint was safety and tolerability
  • Secondary endpoints were hematologic parameters and direct antifibrotic activity


  • N = 18
  • In total, six of ten evaluable patients experienced ≥1 grade reduction in bone marrow fibrosis
    • All six of these patients also experienced stable hemoglobin, white blood cell, and platelet counts
  • After 6 months, one patient experienced spleen volume reduction >35%
    • Another two patients experienced a >50% reduction in Total Symptom Score
    • Overall, one patient experienced an anemia response
  • The safety profile was considered acceptable
    • In total, ten patients discontinued treatment due to adverse events (AEs) or disease progression
    • The most common AEs were gastrointestinal and manageable in most patients


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.

  1.  Topline results from MYLOX-1 trial demonstrate reduction in fibrosis of the bone marrow in patients with myelofibrosis. Published Dec 21, 2023. Accessed Jan 17, 2024.


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