Axitilimab Shows Consistent Efficacy Across Treatment Lines in Chronic Graft-versus-Host Disease
A recent post hoc analysis of the phase 1 AGAVE-201 trial demonstrates that axatilimab, also known as Niktimvo, maintains consistent clinical outcomes for patients with chronic graft-versus-host disease (cGVHD) regardless of the number of prior treatments they have received. The study, presented during the 2025 Transplantation & Cellular Therapy Meetings, highlights the agent’s potential for rapid and durable responses, particularly in patients who follow ruxolitinib therapy.
Overview of the AGAVE-201 Trial
In the AGAVE-201 trial, patients were randomly assigned to receive axatilimab at various doses: 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks, or 3.0 mg/kg every four weeks. The trial included 241 patients in total, offering a comprehensive look at axatilimab’s efficacy across different doses and treatment histories.
Key Findings from the Post Hoc Analysis
The analysis evaluated objective response rates (ORR), time to first response (TTR), sustained response rate (SRR), and organ-specific responses by the number of prior lines of therapy (LOT) and last therapy used. Results showed that axatilimab produced similar ORRs across different LOT categories, despite varying treatment histories.
Response Rates Regardless of Treatment History
Regardless of the best response to their last prior treatment, patients exhibited similar ORRs. Those with complete or partial responses, no change, and progression after last treatments showed ORRs of 64.6%, 62.1%, and 62.5%, respectively. Notably, ORRs slightly increased with the number of prior LOTs, ranging from 51.4% with 2 LOTs to 63.3% with 4 LOTs.
Specific Findings for Axatilimab Doses
The data also revealed dose-specific findings, with varied response rates observed for each dose level. Patients receiving 0.3 mg/kg of axatilimab had ORRs of 63.3%, 81.3%, and 83.3% for complete or partial response, no change, and progression, respectively. Among them, those with 2 LOTs, 3 LOTs, and 4 LOTs had ORRs of 63.6%, 53.3%, and 82.4%, respectively.
Impact of Last Line Therapy
The study highlighted the impact of the last line of therapy on response rates. Patients treated with ruxolitinib in their last LOT had an ORR of 61.8%, a median TTR of 1.9 months, and an SRR of 43.0%. In contrast, those treated with belumosudil had an ORR of 50.0%, a median TTR of 1.9 months, and an SRR of 26.0%.
Organ-Specific Responses
Organ-specific responses were observed irrespective of the last prior therapy. Patients who received rituximab in their last LOT showed higher organ-specific response rates compared to those who received belelmosudil. The observed improvements were noted in various organs, including joints/fascia, mouth, lungs, eyes, and skin.
Demographic Information
The patient population included individuals across a wide age range, with a median age of 53.0 years for all doses and 50.0 years for the 0.3 mg/kg dose. The majority were male, and the number of organs involved at baseline was consistent between groups.
Significance of the Findings
The study underscores the potential of axatilimab for treating steroid-refractory cGVHD, even after multiple lines of therapy. The efficacy demonstrated in patients following ruxolitinib is particularly promising, suggesting a broader application for the agent in managing this challenging condition.
Conclusion and Limitations
Overall, axatilimab appears to be an effective treatment option for patients with chronic graft-versus-host disease, offering consistent and durable responses across various treatment histories and organ involvement. While the findings are encouraging, the small patient sample and varied treatment regimens may limit the generalizability of the results. Nonetheless, these insights could pave the way for personalized treatment approaches in the future.
In light of these findings, the clinical community can explore further avenues for leveraging axatilimab in the management of cGVHD, benefiting a broader patient population.
Next Steps
For healthcare providers and patients, the next step is to closely monitor how axatilimab performs in clinical practice. Ongoing studies and real-world evidence will provide further insights and help refine treatment protocols.
Call to Action
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