Incyte Announces U.S. Food and Drug Administration Grants Priority Review for Axatilimab for the Treatment of Chronic Graft-Versus-Host Disease
February 27 2024 - 4:08PM
Business Wire
- Priority Review acceptance based on positive
results of AGAVE-201 study
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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