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The AGAVE-201 trial: Safety and efficacy of axatilimab at three different doses

Jan 31, 2024
Learning objective: After reading this article, learners will be able to cite a new clinical development in chronic graft-versus-host disease.

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During the 65th American Society of Hematology (ASH) Annual Meeting and Exposition, Wolff presented results from the phase II AGAVE-201 (NCT04710576) trial of axatilimab, an investigational anti-colony-stimulating factor 1 receptor antibody, for the treatment of chronic graft-versus-host disease (cGvHD).1 We summarize the key findings below.

The GvHD hub previously reported positive topline results from the AGAVE-201 trial, indicating the primary endpoint was achieved.

Study design1

In this study, 241 patients (≥2 years) with recurrent or refractory cGvHD, who had received two or more prior lines of therapy, were divided into three cohorts in which they received axatilimab, intravenously, at either 0.3 mg/kg every 2 weeks, 1 mg/kg every 2 weeks or 3 mg/kg every 4 weeks. Patients were permitted to use concomitant medications such as corticosteroids (65%), calcineurin inhibitors (28%), or mTOR inhibitors (12%) but were prohibited from receiving supplementary systemic cGvHD therapy.  

The primary endpoint was the overall response rate at 24 weeks (6 cycles) as defined by the National Institute of Health (NIH) 2014 consensus criteria. The key efficacy endpoint was the proportion of patients reporting a clinically significant reduction of symptoms, measured using the modified Lee Symptom Scale score. Safety endpoints included the frequency and severity of adverse events (AEs).

Efficacy outcomes1

The primary endpoint of overall response rate was met across all cohorts. Figure 1 illustrates efficacy endpoint data within each dose cohort.

Figure 1. Efficacy outcomes of axatilimab* 

ORR, overall response rate; DOR, duration of response; mLSS, modified Lee Symptom Scale; Q2W, every 2 weeks; Q4W, every 4 weeks.
*Adapted from Wolff.1

Safety outcomes1

Three cytomegalovirus infections were reported in the higher-dose cohorts. Table 1 summarizes AEs that occurred in each cohort.

Table 1. Safety results of axatilimab*


0.3 mg/kg
Q2W (%)

1 mg/kg
Q2W (%)

3 mg/kg
Q4W (%)

Drug discontinuation due to TEAEs




Fatal AEs




Most frequent Grade ≥3 TRAEs

              Blood creatine phosphokinase increase




              Periorbital edema








              Gamma-glutamyl transferase increase




Q2W, every 2 weeks; Q4W, every 4 weeks; TEAE, treatment-emergent adverse event; TRAE, treatment-related adverse event.
*Adapted from Wolff.1 and Wolff, et al.2


Data presented from the AGAVE-201 study indicates that axatilimab, when administered at a dose of 0.3 mg/kg every 2 weeks, is an effective treatment option with a manageable safety profile for patients with recurrent or refractory cGvHD.1 A notable reduction in symptom burden was reported by most patients, with AEs being largely of low grade and reversible in most cases.

  1. Wolff D. Safety and efficacy of axatilimab at 3 different doses in patients with chronic graft-versus-host disease (AGAVE-201). Plenary scientific session. 65th American Society of Hematology Annual Meeting and Exposition; Dec 10, 2023; San Diego, US.
  2. Wolff D, Cutler C, Lee S, et al. Safety and efficacy of axatilimab at 3 different doses in patients with chronic graft-versus-host disease (AGAVE-201). Blood. 2023;142(1). DOI: 1182/blood-2023-186963


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