Syndax, Incyte Join Forces to Develop Axatilimab for IPF

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by Marisa Wexler MS |

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Syndax Pharmaceuticals and Incyte are forming a global partnership to develop and commercialize axatilimab, an investigational therapy for idiopathic pulmonary fibrosis (IPF) and other diseases characterized by excessive scarring.

The companies are planning to launch a proof-of-concept Phase 2 clinical trial to test axatilimab in IPF patients early next year.

“We are excited to partner with Syndax and for the opportunity to bring another potential treatment to patients with life-threatening conditions,” Hervé Hoppenot, CEO of Incyte, said in a press release. “Collaborations between companies like Incyte and Syndax, who are both dedicated to scientific advancement, contribute to the development of new innovative medicines that may benefit patient communities around the world.”

Axatilimab is an antibody-based therapy that works by blocking the activity of a protein receptor called colony stimulating factor-1 receptor (CSF-1R).

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Activating this receptor is important for the survival and function of monocytes and macrophages, which are two types of immune cells that are thought to be drivers of IPF and other diseases characterized by excessive tissue scarring (fibrosis). By blocking CSF-1R, axatilimab may lessen these cells’ activitities and, therefore, reduce fibrosis.

In addition to IPF, axatilimab also is being developed to treat chronic graft versus host disease (cGVHD), a serious and potentially fatal complication of stem cell transplants, in which transplanted cells attack healthy tissue in the recipient’s body. This inflammatory attack can lead to the buildup of scar tissue in various organs in the body.

Syndax is currently sponsoring two clinical trials — a Phase 1/2 study (NCT03604692) and a Phase 2 study  (NCT04710576) — to evaluate axatilimab in people with active cGVHD.

Early data from the Phase 1/2 trial indicated the treatment was generally safe and well-tolerated. It also was able to elicit deep and sustainable responses leading to clinically meaningful benefits in several internal organs in patients who were no longer responding to other treatments. Top-line data from the Phase 2 study, called AGAVE-201, is expected in 2023.

Incyte and Syndax now plan to launch additional clinical trials of axatilimab in cGVHD in the near future. The companies also intend to initiate a Phase 2 proof-of-concept trial to test axatilimab in people with IPF early next year.

The U.S. Food and Drug Administration previously gave axatilimab orphan drug designation as a potential therapy for both cGVHD and IPF.

Under the terms of the new collaboration agreement, which must be approved by U.S. antitrust authorities, Incyte will lead global commercial activities for axatilimab across all indications. The companies will share costs of developing the medicine, as well as any profits from its eventual sale.

Syndax also will receive a $117 million upfront payment plus a $35 million investment. The company will be eligible to receive up to $450 million once certain commercial, regulatory, and developmental milestones are met.

“This partnership has the potential to significantly expand and maximize the axatilimab program across multiple lines of treatment in chronic graft-versus-host Disease (cGVHD), as well as additional indications in which the monocyte-macrophage lineage plays a vital role in the fibrotic disease process, such as idiopathic pulmonary fibrosis (IPF),” said Briggs W. Morrison, MD, CEO of Syndax.