Incyte Announces U.S. Food and Drug Administration Grants Priority Review for Axatilimab for the Treatment of Chronic Graft-Versus-Host Disease

Rhea-AI Summary

Incyte's axatilimab receives Priority Review acceptance for chronic GVHD treatment after positive AGAVE-201 trial results, showing promising efficacy and safety.

Insights

Medical Research Analyst

The acceptance of the Biologics License Application (BLA) for axatilimab by the FDA under Priority Review signifies a potentially expedited regulatory pathway. Axatilimab's development targets chronic graft-versus-host disease (GVHD), a condition with limited treatment options after the failure of systemic therapies. Chronic GVHD is a serious complication that can occur after stem cell or bone marrow transplantation, where the donor's immune cells attack the recipient's body, leading to a range of symptoms and affecting quality of life.

The clinical relevance of axatilimab lies in its mechanism of action, which involves the inhibition of the Colony-Stimulating Factor 1 Receptor (CSF-1R). CSF-1R is a critical component in the regulation of macrophages, which are implicated in the inflammatory processes of GVHD. By targeting CSF-1R, axatilimab may mitigate the aberrant immune response characteristic of GVHD. The positive data from the AGAVE-201 trial, demonstrating meaningful clinical results and tolerability, suggests that axatilimab could potentially fill a significant treatment gap for patients.

Financial Analyst

The FDA's Priority Review for axatilimab can be seen as a positive indicator for Incyte and its partnership with Syndax Pharmaceuticals. This expedited review could lead to a faster time-to-market, assuming approval, which is critical in the competitive pharmaceutical landscape. The PDUFA date set for August 28, 2024, provides a clear timeline for investors and analysts to anticipate potential market impact. Moreover, the successful development and potential approval of axatilimab could open additional revenue streams for both companies, especially considering the high unmet medical need and the possibility of axatilimab being a first-in-class therapy for this indication.

Investors should also note the implications of the co-development and co-commercialization agreement between Incyte and Syndax. The terms of such partnerships, including profit-sharing and milestone payments, can significantly affect financial projections and valuations for the involved parties. The market will closely observe any further announcements regarding efficacy, safety and potential market size, which will refine the economic model surrounding axatilimab.

Healthcare Industry Analyst

The Priority Review status granted to axatilimab underscores the FDA's recognition of the urgent need for new treatments in the chronic GVHD space. The current therapeutic landscape for chronic GVHD is characterized by a limited number of FDA-approved drugs, which often have a narrow efficacy profile and significant side effects. Axatilimab's novel approach could disrupt the market by providing a new line of therapy with a different safety and efficacy profile.

For stakeholders in the healthcare industry, including payers and providers, the introduction of axatilimab may lead to changes in treatment protocols and cost structures. Given the complexity and cost of managing chronic GVHD, a new therapy that can demonstrate improved outcomes or reduced side effects could be favorably incorporated into treatment regimens. This could also influence insurance coverage decisions and reimbursement rates, ultimately impacting patient access to the drug.

- Priority Review acceptance based on positive results of AGAVE-201 study

WILMINGTON, Del.--(BUSINESS WIRE)-- Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for axatilimab, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The Prescription Drug User Fee Act (PDUFA) date for the FDA decision is August 28, 2024.

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

Axatilimab is being developed by Incyte and Syndax Pharmaceuticals (Nasdaq:SNDX) as part of an exclusive worldwide co-development and co-commercialization license agreement.

“Despite recent advancements in the treatment of patients with chronic GVHD, there remains a significant unmet need for patients who progressed on earlier lines of therapy,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “Axatilimab’s novel mechanism offers a differentiated treatment approach which may 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 application for axatilimab for this indication.”

The FDA grants Priority Review designation to applications for medicines that, if approved, would treat a serious condition and provide significant improvements in the safety or effectiveness of the treatment.

About Chronic Graft-Versus-Host Disease

Chronic graft-versus-host disease (GVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.^1,2. Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue³.

About Axatilimab

Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and idiopathic pulmonary fibrosis (IPF). Phase 1/2 data of axatilimab in chronic GVHD demonstrating its broad activity and tolerability were last presented at the 63rd American Society of Hematology Annual Meeting and data were published in the Journal of Clinical Oncology. Additionally, positive topline results and additional data from the Phase 2 AGAVE-201 trial highlighted in a Plenary Scientific Session at the American Association of Hematology Annual Meeting 2023 were announced. Axatilimab was granted Orphan Drug Designation by the U.S. FDA for the treatment of patients with chronic GVHD and IPF.

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

About AGAVE-201 (NCT04710576)

The global Phase 2 AGAVE-201 dose-ranging 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 prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct 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. The trial's primary endpoint is the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints include duration of response, percent reduction in daily steroids dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale.

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

About Incyte

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.

Incyte Forward-looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the AGAVE-201 trial, expectations regarding the BLA for axatilimab, and the potential for axatilimab to become a treatment option for chronic graft-versus-host disease, contain predictions, estimates and other forward-looking statements.

These forward-looking statements are based on Incyte's current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the U.S. FDA and other regulatory authorities outside of the United States; the efficacy or safety of Incyte and its partners' products; the acceptance of Incyte and its partners' products in the marketplace; market competition; 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 on form 10-K for the year ended December 31, 2023. Incyte disclaims any intent 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 GVHD Expert Interviews N=32 interviews.

 

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FAQ

What is the significance of the FDA accepting the BLA for axatilimab?

The FDA accepting the BLA for axatilimab for Priority Review indicates that it may offer significant improvements in the treatment of chronic GVHD after positive results from the AGAVE-201 trial.

What is the PDUFA date for the FDA decision on axatilimab?

The Prescription Drug User Fee Act (PDUFA) date for the FDA decision on axatilimab is August 28, 2024.

Who is developing axatilimab for chronic GVHD treatment?

Axatilimab is being developed by Incyte and Syndax Pharmaceuticals as part of an exclusive worldwide co-development and co-commercialization license agreement.

What was highlighted about axatilimab in the AGAVE-201 trial results?

The AGAVE-201 trial results highlighted that treatment with axatilimab resulted in clinically meaningful outcomes, was well-tolerated, and had a manageable safety profile consistent with its mechanism of action.

Why is there a significant unmet need for patients with chronic GVHD?

Despite recent advancements, there is still a significant unmet need for patients with chronic GVHD who have not responded to earlier lines of therapy.