Syndax and Incyte Announce Positive Topline Results from the Pivotal AGAVE-201 Trial of Axatilimab in Chronic Graft-Versus-Host Disease | Syndax Pharmaceuticals, Inc.

Source: Syndax Pharmaceuticals, Press Release 24 July 23

Syndax Pharmaceuticals and Incyte have announced positive topline data from the AGAVE-201 trial of axatilimab, an anti-CSF-1R antibody, in patients with chronic graft-versus-host disease (GVHD) who have undergone two or more prior lines of therapy. The trial achieved its primary endpoint, with overall response rates ranging from 50% to 74% within the first six months of treatment, depending on the dosage. Responses were observed in patients with prior exposure to various therapies.

Based on the promising results, Syndax and Incyte plan to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of 2023. Axatilimab is the first investigational chronic GVHD treatment targeting inflammation and fibrosis through the inhibition of disease-associated macrophages.

The AGAVE-201 study enrolled 241 patients across 16 countries, with a median of four prior systemic therapies. The most common adverse events were consistent with CSF-1R inhibition, and serious adverse events occurred in a subset of patients.

The data has been well-received by the medical community, as chronic GVHD is a significant complication post-allogeneic hematopoietic stem cell transplant, and effective treatment options are urgently needed. The positive results suggest that axatilimab could offer a valuable therapeutic option for patients suffering from this condition.

About graft versus host disease
Chronic graft-versus-host disease (GVHD) is a severe complication of allogeneic hematopoietic stem cell transplantation, where the donor's immune cells attack the recipient's tissues. This condition, which can last for years, affects around 40% of transplant recipients and approximately 14,000 patients in the U.S. Chronic GVHD commonly affects the skin and mucosa and is characterized by the development of fibrotic tissue in multiple organ systems. It is a serious and potentially life-threatening condition.