Incyte and Syndax: Niktimvo led to significant disease response rates in patients with recurrent chronic graft-versus-host-disease (cGVHD).

1. All three tested Niktimvo dosing regimens led to statistically significant overall response rates in patients who have undergone allogenic hematopoietic stem cell transplant and had recurrent or refractory cGVHD.

2. The lowest dosing regimen of 0.3mg/kg every 2 weeks had the highest overall response rate and lowest toxicity.

The Latest

In the international AGAVE-201 study, which was a phase 2, open-label, randomized, multicenter study, 239 patients who underwent allogeneic hematopoietic cell transplant (alloHCT) and had recurrent or refractory chronic graft-versus-host disease (cGVHD) were randomized 1:1:1 to 3 different dosing strategies of Niktimvo. The primary endpoint was evaluated at 24 weeks and all three-dosing groups had statistically significant overall response rates ranging from 50-74%. 53-60% of patients were found to have maintained the response at 12 months. The rate of adverse events leading to drug discontinuation was 6-22%. The lowest dosing regimen of 0.3mg/kg every 2 weeks had the highest overall response rate and lowest toxicity.

Physician’s Perspective

Chronic GVHD is a common complication affecting 30-50% of patients after alloHCT. Chronic GVHD is characterized by inflammatory and fibrotic changes in multiple organs, resulting in high morbidity and mortality. Currently, 20% of all transplanted patients fail primary treatment as well as subsequent trials of the current 3 agents available for cGVHD which are all protein kinase inhibitors. Therefore, novel therapeutics that target alternative pathways implicated in cGVHD are much needed.

Molecular Target of Therapy

Monocytes and macrophages are immune cells that play a key role in the pathogenesis of cGVHD and depend on signaling through Colony-stimulating factor 1 receptor (CSF-1R). Niktimvo (axatilimab-csfr) is a monoclonal antibody that binds to and blocks signaling through CSF-1R, curtailing inflammation and fibrosis that underlie cGVHD.

Company History

Incyte is an American multinational pharmaceutical company focused on developing therapeutics for chronic GVHD, dermatologic disease, and oncologic diseases. Incyte has also developed Jakafi, one of the three currently approved protein kinase inhibitors used to treat cGVHD. Syndax is a clinical-stage biopharmaceutical company focused on cancer therapies. The FDA recently approved Niktimvo at 0.3mg/kg every 2 weeks for the treatment of cGVHD in patients who have failed at least two prior lines of systemic therapies.

Further reading: https://ashpublications.org/blood/article/142/Supplement%201/1/499054/Safety-and-Efficacy-of-Axatilimab-at-3-Different

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