Incyte (Nasdaq: INCY) today announced that the U.S. Food and Drug Administration (FDA) has agreed to subject the Biologics License Application (BIA) to priority review. License Application, BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after the failure of at least two previous lines of systemic treatment. The FDA's decision date under the Prescription Drug User Fee Act (PDUFA) is August 28, 2024.

The BLA builds on positive data from the AGAVE-201 trial (NCT04710576), recently presented during a Plenary Scientific Session at the 2023 American Society of Hematology Annual Meeting, which showed that axatilimab treatment provided clinically meaningful results and was generally well tolerated, with a manageable safety profile consistent with the mechanism of action of CSF-1R inhibition.

Axatilimab is currently being developed by Incyte and Syndax Pharmaceuticals (Nasdaq: SNDX) under an exclusive worldwide co-development and co-commercialization licensing agreement.

“ Despite recent advances in the treatment of patients with chronic GVHD, a significant unmet need remains for patients whose disease has progressed despite previous lines of treatment,” said Hervé Hoppenot, CEO of Incyte. “ The novel mechanism of axatilimab offers a differentiated therapeutic approach that could help patients suffering from this devastating disease. We look forward to working closely with the FDA and our partners at Syndax on the review of our marketing authorization application for axatilimab for this indication. »

The FDA grants priority review status to drug applications that, if approved, could treat a serious disease and provide significant improvements in safety or health. effectiveness of treatment.

About chronic graft-versus-host disease

Chronic graft-versus-host disease (GVHD), an immune response of donor-derived hematopoietic cells against recipient tissues, is a serious, life-threatening complication of hematopoietic stem cell transplantation allogenic, and which can extend over years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients and affects approximately 14,000 patients in the United States 1,2 . Chronic GVHD typically manifests in multiple organ systems, with skin and mucous membranes commonly affected, and is characterized by the development of fibrotic tissue 3 .

About axatilimab

Axatilimab is an investigational monoclonal antibody that targets the colony-stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In preclinical models, inhibition of CSF-1 receptor signaling has been shown to reduce the number of pathogenic macrophages and their monocytic precursors, which play a key role in the fibrosis process underlying diseases such as chronic graft-versus-host disease (GVHD) and idiopathic pulmonary fibrosis (IPF). Phase 1/2 data of axatilimab in chronic GVHD demonstrating its broad activity and tolerability was last presented at the 63rd Annual Meeting of the American Society of Hematology, and the data was published in the Journal of Clinical Oncology. Additionally, positive preliminary results and additional data from the Phase 2 AGAVE-201 trial, presented during the Plenary Scientific Session at the 2023 American Association of Hematology Annual Meeting, were announced. Axatilimab has received orphan drug designation from the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF.

In September 2021, Syndax and Incyte entered into an exclusive global co-development and co-commercialization licensing agreement for axatilimab. Axatilimab is being developed under an exclusive global license from UCB entered into between Syndax and UCB in 2016.

About the AGAVE-201 trial (NCT04710576)

The AGAVE-201 global phase 2 dosing trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD whose disease had progressed after two treatments previous ones. Patients were randomly assigned to one of three treatment groups, where a separate dose of axatilimab administered at 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks or 3.0 mg/kg every four weeks, has been studied. The primary endpoint of the trial was the proportion of patients in each dose group who achieved an objective response, as defined by the 2014 NIH consensus criteria for chronic GVHD, at cycle 7 on day 1. Secondary endpoints included duration of response, percent reduction in daily steroid dose, organ-specific response rates, and validated quality of life assessments using the modified version of the Lee Symptom Scale.

For more information about the AGAVE-201 trial, visit https://www.clinicaltrials.gov/study/NCT04710576 .

About Incyte

Incyte is a global biopharmaceutical company based in Wilmington, Delaware, whose mission is to find solutions to major, unmet medical needs through the discovery, development and commercialization of proprietary therapeutic products. For more information about the company, please visit Incyte.com and follow @Incyte .

Incyte Forward-Looking Statements

Except for historical information discussed herein, information in this press release, including statements regarding the AGAVE-201 trial, expectations regarding submission of a BLA for axatilimab by end of 2023 and the potential for axatilimab to become a therapeutic option for chronic graft-versus-host disease, contains predictions, estimates and other forward-looking statements.

These forward-looking statements are based on Incyte's current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including unanticipated developments and various risk factors such as the following: unforeseen delays; the possibility that continued research and development and clinical trial results may yield unsuccessful or insufficient results to meet applicable regulatory standards or justify continued development; the ability to enroll a sufficient number of subjects in clinical trials; decisions made by the U.S. FDA and other regulatory authorities outside the United States; the effectiveness or safety of Incyte and its partners' products; market acceptance of Incyte and its partners' products; competition in the market; sales, marketing, manufacturing and distribution requirements; and other risks detailed from time to time in Incyte's reports filed with the Securities and Exchange Commission, including its Annual Report and Quarterly Report on Form 10-Q for the quarter ended December 31, 2023. Incyte disclaims any intention or obligation to update these forward-looking statements.

1. SmartAnalyst 2020 SmartImmunology Insights chronic GVHD report.
2. Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis.
3. Kantar 2020 Interviews with GVHD experts N=32 interviews.

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Source: businesswire.com

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