- Trial met its primary endpoint across all
dose cohorts with 74% of patients at the 0.3 mg/kg dose achieving a
complete or partial response within the first six months of
treatment
- Data are featured in the Plenary Scientific
Session at the 65th American Society of Hematology Annual Meeting
2023
- Incyte and its partner Syndax expect to file
a Biologics License Application (BLA) for axatilimab by year-end
2023
Incyte (Nasdaq:INCY) and Syndax Pharmaceuticals (Nasdaq:SNDX)
today announced the full results from the pivotal Phase 2 AGAVE-201
trial of axatilimab, an anti-CSF-1R antibody, in adult and
pediatric patients with refractory chronic graft-versus-host
disease (GVHD) who had received at least two prior lines of
systemic therapy. These data are featured today in the Plenary
Scientific Session (Abstract #1) at the 65th American Society of
Hematology Annual Meeting 2023 (ASH 2023), held December 9-12,
2023, in San Diego and virtually.
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The results, which build on previously announced topline data,
show that the trial met the primary endpoint across all cohorts
receiving axatilimab, at doses of 0.3 mg/kg every two weeks, 1.0
mg/kg every two weeks and 3.0 mg/kg every four weeks. Patients who
received axatilimab at 0.3 mg/kg every two weeks achieved the
highest overall response rate (ORR) of 74% within the first six
months of treatment (95% CI; 63-83). Patients in this cohort
experienced a median time to response to axatilimab of 1.7 months
(0.9-8.1), and 60% of patients maintained a response at 12 months
(measured from first response to new systemic therapy or death,
based on the Kaplan Meier estimate). The recommended dose of
axatilimab for future trials in chronic GVHD is 0.3 mg/kg every two
weeks.
“The data presented today at ASH represent a significant step
forward in expanding the treatment options for patients with
refractory chronic GVHD,” said Pablo J. Cagnoni, M.D., President
and Head of Research and Development, Incyte. "An unmet need
remains for treatments that are well tolerated and efficacious for
patients with refractory chronic GVHD, and the data presented today
show that axatilimab could provide a valuable option. We look
forward to working with our partners at Syndax as we move
axatilimab towards regulatory filing.”
The AGAVE-201 trial also met key secondary endpoints in the 0.3
mg/kg dose, with 55% of patients achieving a ≥7-point improvement
in the modified Lee Symptom Scale (mLSS) score. Organ-specific
responses, including complete responses (CRs), were seen across all
organs involved at baseline, including lower gastrointestinal (GI),
upper GI, esophagus, joints/fascia, mouth, lungs, liver, eyes and
skin. Additionally, responses were notable in fibrosis-dominated
organs, including the esophagus (78%), joints and fascia (76%),
lungs (47%) and skin (27%).
“The additional positive data from AGAVE-201 further strengthen
axatilimab’s strong safety and efficacy profile as a
well-differentiated treatment option for patients with refractory
chronic GVHD,” said Michael A. Metzger, Chief Executive Officer of
Syndax. "As a potentially first-in-class anti-CSF-1R antibody
targeting inflammation and fibrosis through the inhibition of
disease associated macrophages, we have more conviction than ever
that axatilimab is poised to transform the treatment paradigm for
chronic GVHD. Axatilimab has the potential to positively impact
patients with this devastating disease and we are working
diligently with Incyte to bring this agent to market.”
The AGAVE-201 pivotal trial enrolled 241 patients with relapsed
and refractory cGVHD who had received two or more prior systemic
therapies, with 74% having previously received ruxolitinib, 31%
having previously received ibrutinib and 23% having previously
received belumosudil. Patients were enrolled across 121 sites in 16
countries.
The most common treatment-emergent adverse events (TEAEs) were
consistent with the on-target effects of CSF-1R inhibition and with
what was previously observed with axatilimab treatment. TEAEs in
greater than 20% of patients in the overall population (n=239)
include increases in aspartate aminotransferase, blood creatine
phosphokinase, lipase, lactate dehydrogenase, and alanine
aminotransferase.
In the overall trial population, 33% of patients experienced at
least one grade ≥3 TEAE, with 15.5% experiencing adverse events
leading to discontinuation of treatment. For patients who received
axatilimab at 0.3 mg/kg (n=79), grade ≥3 TEAEs occurred in 17.7% of
patients, with 6.3% experiencing TEAEs leading to discontinuation
of treatment.
“Approximately 50% of chronic GVHD patients are refractory to
first-line treatment and 25% of patients require at least four
lines of treatment, representing a great need for additional
effective treatment options,” said Daniel Wolff, M.D., Ph.D., Head,
Senior Physician, and Professor at University Hospital Regensburg.
"Full results from the AGAVE-201 trial show rapid durable responses
documented in all organs and patient subgroups, with significant
symptom burden reduction reported by most of these
heavily-pretreated patients. I am pleased that the results of the
AGAVE-201 trial showed potential advances for patients who had not
responded to previous lines of treatments and look forward to
further research to underscore the efficacy of axatilimab patients
with chronic GVHD.”
Based on these results and pending agreement from the U.S. Food
and Drug Administration (FDA), Syndax and Incyte expect to submit a
Biologics License Application (BLA) to the FDA by year-end
2023.
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
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 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 from the Phase 2 AGAVE-201 trial showing
the trial met its primary endpoint were recently announced.
Axatilimab was granted Orphan Drug Designation by 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 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
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.
About Syndax
Syndax Pharmaceuticals is a clinical stage biopharmaceutical
company developing an innovative pipeline of cancer therapies.
Highlights of the Company's pipeline include revumenib, a highly
selective inhibitor of the Menin–KMT2A binding interaction, and
axatilimab, a monoclonal antibody that blocks the colony
stimulating factor 1 (CSF-1) receptor. For more information, please
visit www.syndax.com or follow the Company on Twitter and
LinkedIn.
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
submission of a BLA for axatilimab by year-end 2023, 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 and
its quarterly report on Form 10-Q for the quarter ended September
30, 2023.
Syndax Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Words such as "may," "will," "expect," "plan," "anticipate,"
"estimate," "intend," "believe" and similar expressions (as well as
other words or expressions referencing future events, conditions or
circumstances) are intended to identify forward-looking statements.
These forward-looking statements are based on Syndax's expectations
and assumptions as of the date of this press release. Each of these
forward-looking statements involves risks and uncertainties. Actual
results may differ materially from these forward-looking
statements. Forward-looking statements contained in this press
release include, but are not limited to, statements about the
progress, timing, clinical development and scope of clinical
trials, the reporting of clinical data for Syndax's product
candidates, the potential filing of a BLA by year-end 2023, and the
potential use of our product candidates to treat various cancer
indications and fibrotic diseases. Many factors may cause
differences between current expectations and actual results,
including: unexpected safety or efficacy data observed during
preclinical or clinical trials; clinical trial site activation or
enrollment rates that are lower than expected; changes in expected
or existing competition; changes in the regulatory environment;
failure of Syndax's collaborators to support or advance
collaborations or product candidates; and unexpected litigation or
other disputes. Other factors that may cause Syndax's actual
results to differ from those expressed or implied in the
forward-looking statements in this press release are discussed in
Syndax's filings with the U.S. Securities and Exchange Commission,
including the "Risk Factors" sections contained therein. Except as
required by law, Syndax assumes no obligation to update any
forward-looking statements contained herein to reflect any change
in expectations, even as new information becomes available.
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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Incyte Contacts:
Media media@incyte.com Investors ir@incyte.com
Syndax Contact: Sharon
Klahre sklahre@syndax.com Tel 781.684.9827
Syndax Pharmaceuticals (NASDAQ:SNDX)
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