Incyte (Nasdaq:INCY) has disclosed that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for Priority Review. This application targets the treatment of chronic graft-versus-host disease (GVHD) following the failure of at least two prior lines of systemic therapy. The FDA's decision is expected by August 28, 2024, under the Prescription Drug User Fee Act (PDUFA).

The BLA submission is supported by positive data from the AGAVE-201 trial (NCT04710576), showcased at the Plenary Scientific Session during the American Society of Hematology Annual Meeting 2023. Results from this trial demonstrated clinically significant outcomes with axatilimab, alongside manageable safety profiles consistent with its mechanism of action in inhibiting CSF-1R.

Axatilimab is a product of collaboration between Incyte and Syndax Pharmaceuticals (Nasdaq:SNDX) under an exclusive worldwide co-development and co-commercialization license agreement.

Incyte, emphasized the pressing need for effective treatments for chronic GVHD, especially for patients who have exhausted existing therapy options. He highlighted axatilimab's novel mechanism of action as offering potential relief for patients facing this challenging condition.

The FDA's Priority Review designation underscores the seriousness of chronic GVHD and the potential of axatilimab to address this unmet medical need effectively.

Chronic graft-versus-host disease (GVHD) arises as an immune reaction from donor-derived cells against recipient tissues following allogeneic hematopoietic stem cell transplantation. It's a severe and potentially life-threatening complication affecting approximately 40% of transplant recipients in the U.S., with around 14,000 patients impacted annually.

Axatilimab, an investigational monoclonal antibody, targets colony-stimulating factor-1 receptor (CSF-1R), believed to regulate the survival and function of monocytes and macrophages. Pre-clinical models have shown that inhibiting CSF-1R signaling can reduce disease-mediating macrophages, which play a critical role in fibrotic diseases like chronic GVHD and idiopathic pulmonary fibrosis (IPF).

The Phase 2 AGAVE-201 trial evaluated axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD. Results showed promising efficacy and tolerability across various dose groups.

Axatilimab's development is under an exclusive worldwide license agreement between Syndax and UCB, with Incyte joining Syndax in its development and commercialization efforts.

The AGAVE-201 trial's primary endpoint focused on the proportion of patients achieving an objective response by a specified timepoint, with secondary endpoints including duration of response, steroid dose reduction, organ-specific response rates, and quality-of-life assessments.

 

Source: businesswire.com

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