Niktimvo was approved by the FDA for cGVHD after failure of at least 2 prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg, based on positive results from the AGAVE-201 trial.1
Primary endpoint1,3
- ORR by cycle 7, day 1 of treatment. Responses were defined by 2014 NIH consensus criteria
Secondary and exploratory endpoints included2,3
- mLSS and DOR
aSteroid doses must have been stable for at least 2 weeks before cycle 1, day 1. CNI or mTOR inhibitor doses must have been stable for at least 2 weeks before randomization.3
bPatients were randomized to 1 of 3 treatment groups that investigated a distinct dose of Niktimvo administered until disease progression, lack of partial response by 9 months, or unacceptable toxicity.3
Target suboptimal responses with Niktimvo1
72% of patients studied had prior exposure to ruxolitinib or another T- or B-cell–modulating therapy1
| Demographics and baseline characteristics of patients with cGVHD1 | 0.3 mg/kg Q2W (N=79) |
|---|---|
| Median age, years (range) | 50 (7-76) |
| Age ≥65 years, n (%) | 21 (27) |
| Male, n (%) | 46 (58) |
| Race, n (%) | |
| White | 67 (85) |
| Asian | 4 (5) |
| Black | 2 (3) |
| Other | 1 (1) |
| Not reported | 5 (6) |
| Median number of months (range) from cGVHD diagnosis | 47 (4-211) |
| ≥4 organs involved, n (%) | 45 (57) |
| Median number of prior lines of therapy (range) | 4 (2-12) |
| Number of prior lines of therapy, n (%) | |
| 2 | 11 (14) |
| 3 | 14 (18) |
| 4 | 17 (22) |
| ≥5 | 37 (47) |
| Prior cGVHD treatment with ibrutinib, n (%)a | 27 (34) |
| Prior cGVHD treatment with ruxolitinib, n (%)a | 57 (72) |
| Prior cGVHD treatment with belumosudil, n (%)a | 16 (20) |
| Refractory to last therapy, n (%) | 37 (47) |
| Severe cGVHD, n (%) | 63 (80) |
| Median Lee Symptom Scale score at baseline (range) | 24 (4-55) |
aNo systemic cGVHD treatments other than those listed were allowed in the study.1,3
A high percentage of patients enrolled had baseline inflammation and fibrosis across multiple organs3
Organ involvement included difficult-to-treat organs3
Skin: 80%
- 72% had deep sclerotic features6
Lungs: 40%
- 47% had FEV1 ≤39%4
Joints/fascia: 69%
cGVHD=chronic graft-versus-host disease; CNI=calcineurin inhibitor; DOR=duration of response; FEV1=forced expiratory volume in 1 second; IV=intravenous; mLSS=modified Lee Symptom Scale; mTOR=mechanistic target of rapamycin; NIH=National Institutes of Health; ORR=overall response rate; Q2W=every 2 weeks; Q4W=every 4 weeks.
References: 1. Niktimvo Prescribing Information. Wilmington, DE: Incyte Corporation. 2. A study of axatilimab at 3 different doses in participants with chronic graft versus host disease (cGVHD) (AGAVE-201). ClinicalTrials.gov. Updated June 16, 2026. Accessed June 16, 2026. https://clinicaltrials.gov/study/NCT04710576. 3. Wolff D, Cutler C, Lee SJ, et al; for the AGAVE-201 Investigators. Axatilimab in recurrent or refractory chronic graft-versus-host disease. N Engl J Med. 2024;391(11):1002-1014. Supplementary appendix available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2401537. 4. Salhotra A, Bhatt V, Thomas B, Lou S, Radojcic V, DeFilipp Z. Axatilimab in patients with lung chronic graft-versus-host disease and related bronchiolitis obliterans syndrome: results from the AGAVE-201 study. Presented at: American Thoracic Society International Conference; May 17-21, 2025; San Francisco, CA. 5. Salhotra A, Defilipp Z, Hamadani M, et al. Efficacy and safety of axatilimab at 3 different doses in patients with chronic graft-versus-host disease (cGVHD) and related bronchiolitis obliterans syndrome (BOS): results of a pivotal phase 2 study. Presented at: European Respiratory Society (ERS) Congress; September 7-11, 2024; Vienna, Austria. Poster 17. 6. Perez-Simon JA, DeFilipp Z, Lee S, et al. Axatilimab for chronic graft-versus-host disease: responses in fibrosis-dominant organs in AGAVE-201. Presented at: 50th Annual Meeting of the EBMT; April 14-17, 2024; Glasgow, Scotland.
