Niktimvo (axatilimab)
Research Synopsis
- Niktimvo (axatilimab) is a monoclonal antibody that targets CSF-1R, showing effectiveness in treating chronic graft-versus-host disease (cGVHD), particularly after failure of prior systemic therapies.
- A recent Phase I/II trial indicated an overall response rate of 50% by day one of cycle 7 in patients aged over 6 with active cGVHD who had undergone at least two treatments.
- Subsequent studies demonstrated an overall response rate of 74%, 67%, and 50% across various axatilimab dosing groups in a Phase 2 trial involving 241 participants.
- The drug is particularly promising for patients with steroid-refractory GVHD, a condition with limited effective treatment options available.
- A significant portion (82%) of patients showed a response during the initial treatment cycles, indicating rapid therapeutic action.
- Common side effects included temporary lab abnormalities, generally manageable in nature.
- There is a growing acknowledgment in the literature about the importance of CSF-1R blockade as a therapeutic strategy for refractory cases of chronic GVHD.
- Research highlights the necessity for further rigorous trials to solidify the safety, efficacy, and optimal dosing of axatilimab, particularly in larger populations.
- Current treatment options for cGVHD remain limited, and the advancements represented by axatilimab could substantially improve patient outcomes in this challenging area of hematologic treatment.
- Articles suggest that while axatilimab shows promise, ongoing research is essential to establish its long-term effectiveness and safety in the GVHD treatment landscape.
Niktimvo (axatilimab)
Axatilimab for Chronic Graft-Versus-Host Disease After Failure of at Least Two Prior Systemic Therapies: Results of a Phase I/II Study.
London, UK
2 hours ago
1 Received
- Chronic graft-versus-host disease (cGVHD) is a significant issue after allogeneic stem cell transplants, and CSF-1R-dependent macrophages contribute to cGVHD fibrosis; axatilimab, a monoclonal antibody that inhibits CSF-1R signaling, shows promise in treating cGVHD.
- A phase I/II clinical trial evaluated the safety and effectiveness of axatilimab in patients over age 6 with active cGVHD who had undergone at least two previous treatments, with an optimal dosing identified and a primary efficacy endpoint met showing a 50% overall response rate by day one of cycle 7.
- Results indicated that 82% of patients responded during the first six
$100 - $150
Hourly Rate
Novel Pharmacological Treatment Options of Steroid-Refractory Graft-versus-Host Disease.
London, UK
2 hours ago
1 Received
- Graft-versus-host disease (GVHD) is a serious complication of hematopoietic stem cell transplants, with current steroid treatments being ineffective in 30-50% of cases, leading to high mortality rates in steroid-refractory GVHD (SR-GVHD).
- This review analyzes new therapeutic options for SR-GVHD, emphasizing experimental drugs like ruxolitinib, monoclonal antibodies, and other agents that show promise but require more research on their safety and effectiveness.
- While some agents demonstrate potential for improving treatment outcomes in chronic GVHD, further rigorous trials are needed to confirm their efficacy on a larger scale.
$100 - $150
Hourly Rate
Current Approaches for the Prevention and Treatment of Acute and Chronic GVHD.
London, UK
2 hours ago
1 Received
- - Acute graft-versus-host disease (aGVHD) is inflammatory while chronic graft-versus-host disease (cGVHD) has autoimmune and fibrotic characteristics; aGVHD is a major risk factor for developing cGVHD, and both share similar but not identical incidence and risk factors.
- - Standard prevention for cGVHD after HLA-matched allogeneic stem cell transplantation (HSCT) includes Calcineurin Inhibitors (CNI) with Methotrexate (MTX) or Mycophenolate (MMF), while first-line treatments for aGVHD include corticosteroids and Ruxolitinib for second-line therapy.
- - Effective treatments for steroid-refractory cGVHD include R
$100 - $150
Hourly Rate
CSF1R Blockade for Refractory Chronic Graft-versus-Host Disease.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Targeting CSF1R in Chronic GVHD - Lessons in Translation.
London, UK
2 hours ago
1 Received
$100 - $150
Hourly Rate
Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease.
London, UK
2 hours ago
1 Received
- The study investigates the effectiveness of axatilimab, a CSF1R-blocking antibody, for treating chronic graft-versus-host disease (GVHD) in patients post-hematopoietic stem-cell transplantation.
- In a phase 2 trial involving 241 participants, different doses of axatilimab were tested, with overall response rates of 74%, 67%, and 50% across three dose groups.
- Alongside substantial improvement in GVHD symptoms measured by patient-reported outcomes, the most frequent side effects were temporary lab abnormalities linked to the treatment.
$100 - $150
Hourly Rate