Axatilimab Continues to Improve Outcomes in Chronic GVHD

MIcroscopic image of bone marrow cells - Generated with Google Gemini AI

Axatilimab-csfr (Niktimvo) demonstrated high response rates in patients with recurrent or refractory chronic graft-vs-host disease (GVHD), confirming the agent’s promise in this patient population.^1,2

Data from the phase 2 AGAVE-201 study (NCT04710576) recently published in The New England Journal of Medicine reported that in 241 patients with recurrent or refractory GVHD, the overall response rate was 74% (95% CI, 63%-83%) within the first 6 months of treatment at a dose of 0.3 mg/kg every 2 weeks.

The median time to response was 1.7 months (range, 0.9-8.1) in this cohort, and an estimated 60% of patients maintained a response at 12 months. A reduction of symptoms by more than 5 points on the modified Lee Symptom scale was also reported in 60% of patients at the 0.3-mg/kg dose.

"Results from the AGAVE-201 trial show rapid, durable responses in all organs studied and patient subgroups, with clinically meaningful symptom burden reduction reported by most of these heavily pretreated patients who had not responded to previous lines of treatment," said Daniel Wolff, MD, PhD, senior physician professor at University Hospital Regensburg and study author, in a press release.¹ "As patients with chronic GVHD often cycle through the currently available therapies in the pursuit of relief from this debilitating disease, with nearly 50% of patients requiring more than two lines of therapy, I am pleased that [axatiimab] will soon be available for these patients in need."

In August 2024, the FDA approved axatilimab for the treatment of patients with chronic GVHD that experienced failure of at least 2 prior lines of therapy. This approval was supported by data from AGAVE-201.³

Regarding safety, 6.3% of patients in the 0.3-mg/kg cohort had adverse effects (AEs) that led to dose decreases, and 6.3% of patients discontinued treatment due to AEs. The most common AEs of any grade seen in at least 20% of patients in this cohort consisted of fatigue (22.8%), headache (19.0%), periorbital edema (2.5%), and COVID-19 (16.5%).⁴

Laboratory abnormalities observed in the study included increased aspartate aminotransferase (13.9%), increased creatinine phosphokinase (11.4%), increased lipase (11.4%), increased lactate dehydrogenase (13.9%), increased alanine aminotransferase (12.7%), and increased amylase (3.8%). A total of 17.7% of patients had at least 1 related grade 3 or higher AE and 1 patient had an AE that led to death.

REFERENCES:

1. Incyte and Syndax announce New England Journal of Medicine publication of data from pivotal AGAVE-201 Trial of Niktimvo™ (axatilimab-csfr) in chronic graft-versus-host disease. News release. Syndax Pharmaceuticals. September 18, 2024. Accessed September 20, 2024. https://tinyurl.com/4nf7nynu

2. Wolff D, Cutler C, Lee SJ, et al. Axatilimab in recurrent or refractory chronic graft-versus-host disease. New Engl J Med. 2024;391:1002-1014. doi:10.1056/NEJMoa2401537

3. FDA approves axatilimab-csfr for chronic graft-versus-host disease. News release. FDA. August 14, 2024. Accessed September 20, 2024. https://tinyurl.com/3sn66tn3

4. Wolff D, Cutler C, Lee SJ, et al. Safety and efficacy of axatilimab at 3 different doses in patients with chronic graft-versus-host disease (AGAVE-201). Blood. 2023;142(suppl 1):1. doi:10.1182/blood-2023-186963