- Niktimvo™ (axatilimab-csfr) is the first approved anti-CSF-1R
antibody targeting the drivers of inflammation and fibrosis seen in
chronic GVHD
- Pivotal data from the AGAVE-201 study supporting the approval
show treatment with Niktimvo resulted in durable responses across
all organs studied and patient subgroups
- Syndax conference call and webcast scheduled for today at 6:00
p.m. ET
Incyte (Nasdaq:INCY) and Syndax Pharmaceuticals (Nasdaq:SNDX)
today announced that the U.S. Food and Drug Administration (FDA)
has approved Niktimvo™ (axatilimab-csfr), 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 in adult
and pediatric patients weighing at least 40 kg (88.2 lbs.).
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“With the approval of Niktimvo, patients with chronic GVHD whose
disease has progressed after prior therapies, now have a new
treatment option with a novel mechanism of action to help address
the serious and devastating complications associated with this
disease,” said Hervé Hoppenot, Chief Executive Officer, Incyte.
“Niktimvo is Incyte’s second approved treatment for chronic GVHD,
underscoring our continued commitment to advancing the development
of new medicines on behalf of patients with this disease and the
medical community.”
Chronic GVHD is a serious condition that can occur after an
allogeneic stem cell transplant (the transfer of stem cells from a
donor) in which the donated cells initiate an immune response and
attack the transplant recipient’s organs. Chronic GVHD is a leading
cause of significant morbidity and mortality after an allogeneic
stem cell transplant and is estimated to develop in approximately
42% of transplant recipients, affecting approximately 17,000
patients in the U.S.1 Of those patients who develop chronic GVHD,
nearly 50% require at least three lines of treatment, emphasizing
the need for additional effective treatment options.2
“The approval of Niktimvo represents a significant treatment
advancement for patients with chronic GVHD who have failed at least
two lines of previous therapy,” said Michael A. Metzger, Chief
Executive Officer, Syndax. “We look forward to bringing this
first-in-class anti-CSF-1R antibody to patients in need of new
treatment options, while also continuing to explore axatilimab’s
potential in combination with other standard of care therapies for
chronic GVHD and in other indications.”
The FDA approval was based on data from the global AGAVE-201
study evaluating the safety and efficacy of Niktimvo in 241 adult
and pediatric patients with refractory chronic GVHD who received at
least two prior lines of systemic therapy. The trial met the
primary endpoint across all cohorts receiving Niktimvo. Results
from the study showed durable responses across all organs studied
and patient subgroups. Among patients who received Niktimvo at the
approved dose of 0.3 mg/kg every two weeks (N=79), 75% achieved an
overall response rate (ORR) within the first six months of
treatment, with a median time to response of 1.5 months.
Additionally, 60% maintained a response at 12 months (measured from
first response to new systemic therapy or death, based on the
Kaplan Meier estimate). The trial also met a key exploratory
endpoint, with a majority (56%) of patients achieving a ≥7-point
improvement in the modified Lee Symptom Scale (mLSS) score.
Organ-specific complete and partial responses were demonstrated
across all organs studied that are affected by chronic GVHD,
including lower gastrointestinal (GI), upper GI, esophagus,
joints/fascia, mouth, lungs, liver, eyes and skin.
Serious adverse reactions occurred in 44% of patients who
received Niktimvo (N=79). Serious adverse reactions observed in
>2 patients included infection (pathogen unspecified), viral
infection and respiratory failure. Permanent discontinuation of
Niktimvo due to an adverse reaction occurred in 10% of patients and
dose reduction due to adverse reaction occurred in 8% of patients.
Dose interruptions due to an adverse reaction occurred in 44% of
patients. The adverse reactions leading to dose interruption in
>2 patients were viral infection, infection (pathogen
unspecified), bacterial infection, musculoskeletal pain and
pyrexia.
The most common (≥15%) adverse reactions, including laboratory
abnormalities, were increased aspartate aminotransferase (AST),
infection (pathogen unspecified), increased alanine
aminotransferase (ALT), decreased phosphate, decreased hemoglobin,
viral infection, increased gamma glutamyl transferase (GGT),
musculoskeletal pain, increased lipase, fatigue, increased amylase,
increased calcium, increased creatine phosphokinase (CPK),
increased alkaline phosphatase (ALP), nausea, headache, diarrhea,
cough, bacterial infection, pyrexia and dyspnea.
“Advanced chronic GVHD is characterized by the development of
fibrotic tissue across multiple organ systems, including most
commonly the skin and mucosa, and can be extremely difficult to
treat, leading to high rates of morbidity and mortality,” said
Daniel Wolff, M.D., Ph.D., Head of the GVHD Center at the
University Hospital Regensburg. “I am excited that Niktimvo is
designed to specifically target key drivers of inflammation and
fibrosis in chronic GVHD, and I am highly encouraged by the robust
responses observed across all organs and patient subgroups within
the heavily pre-treated population enrolled in the AGAVE-201 trial.
I look forward to having a new and differentiated treatment option
for my patients who need additional therapies to address this very
difficult to manage, debilitating, disease.”
The Biologics License Application (BLA) for Niktimvo for the
treatment of chronic GVHD after failure of at least two prior lines
of systemic therapy in adult and pediatric patients weighing at
least 40 kg (88.2 lbs) was reviewed by the FDA under Priority
Review. 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.
In the United States, Niktimvo will be co-commercialized by
Incyte and Syndax Pharmaceuticals. Incyte has exclusive
commercialization rights for Niktimvo outside of the U.S.
To facilitate patient dosing and limit product waste, following
the FDA’s approval of Niktimvo (as a 50mg vial), Incyte and Syndax
will seek approval to launch two smaller vial sizes. Following FDA
approval of the new vial sizes, the Companies anticipate launching
Niktimvo in the U.S., no later than early first quarter 2025.
Syndax Conference Call Information Syndax will host a
conference call and live audio webcast at 6:00 p.m. ET, today,
August 14, 2024.
The live audio webcast may be accessed through the Events &
Presentations page in the Investors section of the Company’s
website. Alternatively, the conference call may be accessed through
the following:
Domestic Dial-in Number: 1-800-860-2442 International Dial-in
Number: 1-412-858-4600 Please ask to be joined into the Syndax
Pharmaceuticals call. Live webcast:
https://event.choruscall.com/mediaframe/webcast.html?webcastid=6M3ZcewG
For those unable to participate in the conference call or
webcast, a replay will be available on the Investors section of the
Company’s website at www.syndax.com approximately 24 hours after
the conference call and will be available for 90 days following the
call.
About AGAVE-201 The global 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 was 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 Niktimvo™ (axatilimab-csfr) Niktimvo
(axatilimab-csfr) is a first-in-class anti-CSF-1R antibody approved
for use in the U.S. for the treatment of chronic graft-versus-host
disease (cGVHD) after failure of at least two prior lines of
systemic therapy in adult and pediatric patients weighing at least
40 kg (88.2 lbs).
In 2016, Syndax licensed exclusive worldwide rights to develop
and commercialize axatilimab from UCB. In September 2021, Syndax
and Incyte entered into an exclusive worldwide co-development and
co-commercialization license agreement for axatilimab in cGVHD and
any future indications.
Axatilimab is being studied in frontline combination trials in
chronic GVHD – a Phase 2 combination trial with ruxolitinib
(NCT06388564) and a Phase 3 combination trial with steroids are
expected to initiate by year end. Axatilimab is also being studied
in an ongoing Phase 2 trial in patients with idiopathic pulmonary
fibrosis (NCT06132256).
Niktimvo is a trademark of Incyte.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS Infusion-Related
Reactions Niktimvo™ (axatilimab-csfr) can cause
infusion-related reactions. Infusion-related reactions, including
hypersensitivity reactions, occurred in 18% of patients who
received Niktimvo in the clinical trial (AGAVE-201), with Grade 3
or 4 reactions in 1.3%.
Premedicate with an antihistamine and an antipyretic for
patients who have previously experienced an infusion-related
reaction to Niktimvo. Monitor patients for signs and symptoms of
infusion-related reactions, including fever, chills, rash,
flushing, dyspnea, and hypertension. Interrupt or slow the rate of
infusion or permanently discontinue Niktimvo based on severity of
the reaction.
Embryo-Fetal Toxicity Based on its mechanism of action,
Niktimvo may cause fetal harm when administered to a pregnant
woman. Advise pregnant women of the potential risk to the fetus.
Advise females of reproductive potential to use effective
contraception during treatment with Niktimvo and for 30 days after
the last dose.
ADVERSE REACTIONS Serious adverse reactions occurred in
44% of patients who received Niktimvo (N=79). Serious adverse
reactions in >2 patients included infection (pathogen
unspecified), viral infection and respiratory failure. Permanent
discontinuation of Niktimvo due to an adverse reaction occurred in
10% of patients and dose reduction due to adverse reaction occurred
in 8% of patients. Dose interruptions due to an adverse reaction
occurred in 44% of patients. The adverse reactions leading to dose
interruption in >2 patients were viral infection, infection
(pathogen unspecified), bacterial infection, musculoskeletal pain,
and pyrexia.
The most common (≥15%) adverse reactions, including laboratory
abnormalities, were increased aspartate aminotransferase (AST),
infection (pathogen unspecified), increased alanine
aminotransferase (ALT), decreased phosphate, decreased hemoglobin,
viral infection, increased gamma glutamyl transferase (GGT),
musculoskeletal pain, increased lipase, fatigue, increased amylase,
increased calcium, increased creatine phosphokinase (CPK),
increased alkaline phosphatase (ALP), nausea, headache, diarrhea,
cough, bacterial infection, pyrexia, and dyspnea.
Clinically relevant adverse reactions in <10% of patients who
received Niktimvo included:
- Eye disorders: periorbital edema
- Skin and subcutaneous skin disorders: pruritus
- Vascular disorders: hypertension
Immunogenicity: Anti-Drug Antibody–Associated Adverse
Reactions Across treatment arms in patients with cGVHD who
received Niktimvo in clinical trials, among the patients who
developed anti-drug antibodies (ADAs), hypersensitivity reactions
occurred in 26% (13/50) of patients with neutralizing antibodies
(NAb) and in 4% (2/45) of those without NAb.
USE IN SPECIFIC POPULATIONS Lactation Because of
the potential for serious adverse reactions in a breastfed child,
advise women not to breastfeed during treatment and for 30 days
after the last dose of Niktimvo.
Females and Males of Reproductive Potential Pregnancy Testing Verify pregnancy status in
females of reproductive potential prior to initiating Niktimvo.
Contraception Females Advise
females of reproductive potential to use effective contraception
during treatment with Niktimvo and for 30 days after the last dose
of Niktimvo.
DOSAGE AND ADMINISTRATION Dosage Modifications for
Adverse Reactions Monitor aspartate aminotransferase (AST),
alanine aminotransferase (ALT), alkaline phosphatase (ALP),
creatine phosphokinase (CPK), amylase, and lipase prior to the
start of Niktimvo therapy, every 2 weeks for the first month, and
every 1 to 2 months thereafter until abnormalities are resolved.
See Table 1 in the Prescribing Information for more
recommendations.
Please see the full Prescribing Information for Niktimvo.
About Incyte A global biopharmaceutical company on a
mission to Solve On., Incyte follows the science to find solutions
for patients with unmet medical needs. Through the discovery,
development and commercialization of proprietary therapeutics,
Incyte has established a portfolio of first-in-class medicines for
patients and a strong pipeline of products in Oncology and
Inflammation & Autoimmunity. Headquartered in Wilmington,
Delaware, Incyte has operations in North America, Europe and
Asia.
For additional information on Incyte, please visit Incyte.com or
follow us on social media: LinkedIn, X, Instagram, Facebook,
YouTube.
About Syndax Syndax Pharmaceuticals is a commercial stage
biopharmaceutical company developing an innovative pipeline of
cancer therapies. Highlights of the Company's pipeline include
revumenib a highly selective menin inhibitor, and Niktimvo™
(axatilimab-csfr), a monoclonal antibody that blocks the colony
stimulating factor 1 (CSF-1) receptor. Syndax is working to unlock
the full potential of its pipeline and is conducting several
clinical trials across the continuum of treatment for both
revumenib and Niktimvo. For more information, please visit
www.syndax.com/ or follow the Company on X (formerly 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 whether and when
Niktimvo might provide a successful treatment option for patients
with chronic GVHD and statements regarding the potential for
axatilimab to treat other conditions, 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 U.S.; 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 and its report on
form 10-Q for the quarter ended June 30, 2024. Incyte disclaims any
intent or obligation to update these forward-looking
statements.
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 acceptance of Syndax and its partners' products in
the marketplace, sales, marketing, manufacturing and distribution
requirements, 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 Data on file. 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.
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Incyte: Media
media@incyte.com
Investors ir@incyte.com
Syndax: Sharon Klahre
sklahre@syndax.com 781.684.9827
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