Study met primary endpoint, demonstrating a
higher proportion of sustained platelet response with VYVGART
treatment compared to placebo (p=0.0316); responders observed
across patient types regardless of prior therapy or disease
severity
Statistically significant separation from placebo
in key platelet-derived secondary endpoints
Safety and tolerability profile of VYVGART is
consistent with previous clinical trials; ADVANCE is first
registrational trial with chronic dosing out to 24 weeks
Topline data expected in first quarter 2023 from
ADVANCE-SC, the second pivotal trial required for registration in
primary immune thrombocytopenia (ITP)
argenx to host investor call today at 8:30am ET /
2:30pm CET
Regulated Information/Inside
Information
Breda, the
Netherlands—May
5, 2022—argenx
SE (Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases, today announced positive data from the Phase 3
ADVANCE trial of VYVGART® (efgartigimod alfa-fcab) in adults with
primary ITP. ADVANCE met its primary endpoint demonstrating that a
higher proportion of chronic ITP patients receiving VYVGART
achieved a sustained platelet count response compared to placebo.
ADVANCE is the first Phase 3 clinical trial of a neonatal Fc
receptor (FcRn) blocker in ITP.
“Immune thrombocytopenia is a rare, debilitating
autoimmune disease that can be very difficult to treat, especially
in patients who have an insufficient response to previous ITP
therapies. There is no clear standard of care and many patients
continue to experience significant symptoms and decreased quality
of life,” said Catherine Broome, M.D., Associate Professor of
Medicine at Georgetown University Lombardi Comprehensive Cancer
Center, and Principal Investigator in the ADVANCE study. “These
data are very promising as they show that platelet counts can
rapidly improve to clinically meaningful levels following VYVGART
treatment in a proportion of a heavily pretreated patient
population. We are excited that targeting pathogenic IgG
autoantibodies could represent a new, potential approach to help
alleviate the disease burden in this patient community.”
The ADVANCE trial enrolled 131 adult patients
with chronic and persistent ITP. Patients were heavily pretreated
and 67% of patients had received three or more prior ITP therapies,
including 59% who had prior thrombopoietin receptor agonist
(TPO-RAs) experience, 34% with prior rituximab experience and 37%
with a history of splenectomy. Patients were insufficiently
controlled at baseline with mean platelet counts of 17x109/L across
all patients. Of patients who completed the full ADVANCE study, 94%
(63/67) of VYVGART-treated patients and 97% (38/39) of placebo
patients continued to the ADVANCE+ open-label extension study.
Highlights of Phase
3 ADVANCE Data
Primary endpoint met ADVANCE
met its primary endpoint demonstrating a significantly higher
proportion of patients with chronic ITP receiving VYVGART (17/78;
21.8%) compared to placebo (2/40; 5%) achieved a sustained platelet
response (p=0.0316), defined as having platelet counts greater than
or equal to 50x109/L on at least four of the last six scheduled
visits between weeks 19 and 24 of treatment.
Primary endpoint responders were observed across
patient types regardless of age, disease severity, time since
diagnosis, prior ITP treatment or background medication.
Key platelet-derived secondary
endpoints demonstrated
statistical significanceKey platelet-derived
secondary endpoints showed VYVGART-treated patients had a
statistically significant benefit compared to placebo on (1)
cumulative number of weeks where platelet counts were at least
50x109/L in the chronic ITP population (p=0.0009) and (2) sustained
platelet response in the overall population, including both chronic
and persistent ITP patients (p=0.0108). Numerically fewer
WHO-classified bleeding events occurred in treated patients
throughout the trial but the difference from placebo was not
statistically significant. A higher proportion of treated patients
in the overall population achieved a durable, sustained platelet
response compared to placebo, defined as a sustained platelet
response on at least six of the last eight scheduled visits between
weeks 17 and 24 of treatment (p=0.0265), but was not considered
statistically significant based on hierarchical testing.
Additional secondary
endpoints provided
clinically meaningful data on platelet count responses
throughout 24-week trial Additional secondary endpoint
data from the ADVANCE trial are consistent with primary and
secondary platelet-derived endpoints and provide additional context
on metrics that often drive treatment decisions.
- International Working Group
(IWG) responder status: 51.2% of VYVGART-treated patients
were classified as IWG responders and 27.9% as complete responders
compared to 20% of placebo patients as IWG responders and 4.4% as
complete responders. IWG responders are defined as having a
platelet count of at least 30x109/L, a two-fold increase in
platelet count from baseline, and the absence of bleeding for two
separate, consecutive weekly visits. Complete responders are
patients with platelet counts of 100x109/L and the absence of
bleeding for two separate, consecutive weekly visits.
- Mean platelet count change
from baseline: VYVGART-treated patients demonstrated a
rapid onset of platelet count improvement with statistically
significant separation from placebo observed at week one and
maintained through 20 out of 24 weeks of the trial.
- Switch to
biweekly dosing: Ten VYVGART-treated
patients switched to a biweekly (every two weeks) dosing schedule
after achieving platelet counts of 100x109/L for three out of four
consecutive visits, compared to one placebo patient. Nine of the
ten treated patients achieved a sustained platelet response.
Consistent safety and tolerability
profileADVANCE is the second registrational trial of
VYVGART and the first to evaluate chronic weekly dosing. VYVGART
was well-tolerated in this 24-week study and the observed safety
and tolerability profile was consistent with previous clinical
trials.
“In listening to and learning from people in the
ITP community, we understand the impact of living with this disease
can extend beyond physical signs, taking a serious toll on a
person’s quality of life. These compelling preliminary data
emphasize the potential for VYVGART to drive responses in ITP
regardless of prior lines of therapy, history of splenectomy or
time from diagnosis. We look forward to learning more about the
potential approach of targeting pathogenic IgGs in ITP through our
ADVANCE-SC trial, which is on track to read out in the first
quarter of next year,” said Luc Truyen, MD, Ph.D., Chief Medical
Officer at argenx. “The totality of data generated thus far
continue to support the key attributes of VYVGART, including its
onset of action and safety profile. These data further reinforce
our confidence in FcRn blockade as a precision tool with the
potential to reach a broad spectrum of IgG-mediated severe
autoimmune diseases.”
The Phase 3 ADVANCE trial is the first of two
registrational trials being conducted as part of the ongoing ITP
development program. ADVANCE-SC is evaluating subcutaneous
efgartigimod for the treatment of primary ITP. Topline data from
the ADVANCE-SC study are expected in the first quarter of 2023.
Phase 3 ADVANCE Trial Design
The Phase 3 ADVANCE trial was a randomized, double-blind,
placebo-controlled, multicenter, global trial evaluating the
efficacy and safety of VYVGART in adult patients with chronic or
persistent primary ITP. A total of 131 adult patients with primary
ITP in North America, Europe and Japan enrolled in the trial and
received VYVGART or placebo for a total of 24 weeks as part of the
primary trial. Enrolled patients had a confirmed ITP diagnosis and
a mean entry platelet count of less than 30x109/L. Patients were on
a stable dose of at least one ITP treatment prior to randomization
and had received at least one prior therapy. Concomitant
medications permitted included corticosteroids, nonsteroidal
immunosuppressive drugs, fostamatinib or TPO-RAs. If patients were
on 'watch and wait' at baseline, they had to have received at least
2 prior treatments for ITP.
Patients were randomized in a 2:1 ratio to
receive VYVGART or placebo for a total of 24 weeks as part of the
primary trial. Randomized patients received weekly infusions from
weeks 1-4 and were eligible to adjust frequency to bi-weekly
depending on platelet count. Administration frequency was fixed
from study visits 16-24. The primary endpoint was measured by the
proportion of patients with chronic ITP with a sustained platelet
count response defined as achieving platelet counts of greater than
or equal to 50x109/L for at least four of the last six scheduled
visits between weeks 19 and 24. Patients who received rescue
therapy at week 12 or later, or for whom dose and/or frequency of
concurrent ITP therapies increased at week 12 or later, were
considered non-responders. Key secondary endpoints included extent
of disease control over 24-week treatment period, proportion of
overall population with sustained platelet count response,
incidence and severity of WHO-classified bleeding events and an
extended primary endpoint analysis between weeks 17 and 24.
About Immune Thrombocytopenia
(ITP)Immune thrombocytopenia (ITP) is an autoimmune
disorder where immunoglobulin G (IgG) autoantibodies destroy
platelets and reduce platelet production, which can lead to an
increased risk of excessive bleeding and bruising. In severe cases,
frequent bleeding events can cause anemia or even brain hemorrhage
in rare cases. ITP is also associated with debilitating fatigue and
significant impacts on mental health, including anxiety, fear and
depression. Many ITP patients are inadequately controlled on
current therapies so there remains a significant unmet need for
additional treatment options.
About
VYVGART® (efgartigimod
alfa-fcab)
VYVGART is a human IgG1 antibody fragment that
binds to the neonatal Fc receptor (FcRn), resulting in the
reduction of circulating immunoglobulin G (IgG) autoantibodies. It
is the first and only approved FcRn blocker. VYVGART is approved in
the United States for the treatment of adults with generalized
myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR)
antibody positive and in Japan for the treatment of adults with gMG
who do not have sufficient response to steroids or non-steroidal
immunosuppressive therapies (ISTs). VYVGART is being studied in
adults with primary immune thrombocytopenia (ITP) and other IgG
autoantibody-mediated diseases.
Important Safety Information for
VYVGART® (efgartigimod
alfa-fcab) intravenous (IV)
formulation (U.S. prescribing information) for the
treatment of generalized myasthenia gravis (gMG) in adult
patients who are anti-acetylcholine receptor
(AChR) antibody
positive.
What is
VYVGART® (efgartigimod
alfa-fcab)?VYVGART is a
prescription medicine used to treat a condition called generalized
myasthenia gravis, which causes muscles to tire and weaken easily
throughout the body, in adults who are positive for antibodies
directed toward a protein called acetylcholine receptor (anti-AChR
antibody positive).What is the most important information I should
know about VYVGART? VYVGART may cause serious side
effects, including:
-
Infection. VYVGART may increase the risk of
infection. In a clinical study, the most common infections were
urinary tract and respiratory tract infections. More patients on
VYVGART vs placebo had below normal levels for white blood cell
counts, lymphocyte counts, and neutrophil counts. The majority of
infections and blood side effects were mild to moderate in
severity. Your health care provider should check you for infections
before starting treatment, during treatment, and after treatment
with VYVGART. Tell your health care provider if you have any
history of infections. Tell your health care provider right away if
you have signs or symptoms of an infection during treatment with
VYVGART such as fever, chills, frequent and/or painful urination,
cough, pain and blockage of nasal passages/sinus, wheezing,
shortness of breath, fatigue, sore throat, excess phlegm, nasal
discharge, back pain, and/or chest pain.
- Undesirable immune
reactions (hypersensitivity reactions). VYVGART can
cause the immune system to have undesirable reactions such as
rashes, swelling under the skin, and shortness of breath. In
clinical studies, the reactions were mild or moderate and occurred
within 1 hour to 3 weeks of administration, and the reactions did
not lead to VYVGART discontinuation. Your health care provider
should monitor you during and after treatment and discontinue
VYVGART if needed. Tell your health care provider immediately about
any undesirable reactions.
Before taking VYVGART, tell your health care
provider about all of your medical conditions, including if
you:
- Have a history of infection or you
think you have an infection.
- Have received or are scheduled to
receive a vaccine (immunization). Discuss with your health care
provider whether you need to receive age-appropriate immunizations
before initiation of a new treatment cycle with VYVGART. The use of
vaccines during VYVGART treatment has not been studied, and the
safety with live or live-attenuated vaccines is unknown.
Administration of live or live-attenuated vaccines is not
recommended during treatment with VYVGART.
- Are pregnant or plan to become
pregnant and are breastfeeding or plan to breastfeed.
Tell your health care provider about all the
medicines you take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements.
What are the common side effects of
VYVGART?The most common side effects of VYVGART are
respiratory tract infection, headache, and urinary tract
infection.
These are not all the possible side effects of
VYVGART. Call your doctor for medical advice about side effects.
You may report side effects to the US Food and Drug Administration
at 1-800-FDA-1088.
Please see the full Prescribing
Information for VYVGART and talk to your doctor.
About argenx argenx
is a global immunology company committed to improving the lives of
people suffering from severe autoimmune diseases. Partnering with
leading academic researchers through its Immunology Innovation
Program (IIP), argenx aims to translate immunology breakthroughs
into a world-class portfolio of novel antibody-based medicines.
argenx developed and is commercializing the first-and-only approved
neonatal Fc receptor (FcRn) blocker in the U.S. and Japan. The
Company is evaluating efgartigimod in multiple serious autoimmune
diseases and advancing several earlier stage experimental medicines
within its therapeutic franchises. For more information, visit
www.argenx.com and follow us on LinkedIn, Twitter, and
Instagram.
For further information, please
contact: Media:Kelsey
Kirkkkirk@argenx.com
Investors:Beth
DelGiaccobdelgiacco@argenx.com
Michelle Greenblattmgreenblatt@argenx.com
Forward-looking Statements
The contents of this announcement include
statements that are, or may be deemed to be, “forward-looking
statements.” These forward-looking statements can be identified by
the use of forward-looking terminology, including the terms
“believes,” “hope,” “estimates,” “anticipates,” “expects,”
“intends,” “may,” “will,” or “should” and include statements argenx
makes concerning the expected long-term safety, tolerability and
efficacy of VYVGART® (efgartigimod alfa-fcab) in adult patients
with Primary Immune thrombocytopenia. By their nature,
forward-looking statements involve risks and uncertainties and
readers are cautioned that any such forward-looking statements are
not guarantees of future performance. argenx’s actual results may
differ materially from those predicted by the forward-looking
statements as a result of various important factors. A further list
and description of these risks, uncertainties and other risks can
be found in argenx’s U.S. Securities and Exchange Commission (SEC)
filings and reports, including in argenx’s most recent annual
report on Form 20-F filed with the SEC as well as subsequent
filings and reports filed by argenx with the SEC. Given these
uncertainties, the reader is advised not to place any undue
reliance on such forward-looking statements. These forward-looking
statements speak only as of the date of publication of this
document. argenx undertakes no obligation to publicly update or
revise the information in this press release, including any
forward-looking statements, except as may be required by law.
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