ADHERE data show VYVGART®
Hytrulo has potential to be first advancement for CIDP patients in
30 years
Real-world data demonstrate gMG patients able to
significantly reduce steroid use over first six months of
initiating VYVGART® treatment
April 16, 2024 – 7:00am CET
Amsterdam, the Netherlands –
argenx SE (Euronext & Nasdaq: ARGX), a global immunology
company committed to improving the lives of people suffering from
severe autoimmune diseases, today announced that data from its
Phase 3 ADHERE trial evaluating VYVGART Hytrulo (efgartigimod alfa
and hyaluronidase-qvfc) in patients with chronic inflammatory
demyelinating polyneuropathy (CIDP) were presented for the first
time to the medical community during the Clinical Trials Plenary
Session at the American Academy of Neurology (AAN) Annual Meeting
in Denver, CO.
argenx also highlighted clinical trial and
real-world data across seven posters and presentations that
continue to reinforce VYVGART and VYVGART Hytrulo as a
transformative treatment option for gMG patients.
“Our innovative approach to autoimmunity
research is changing expectations for the global immunology
community,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer,
argenx. “The gMG and CIDP data presented at AAN reinforce that our
pioneering approach to transforming autoimmunity is redefining what
is possible for patients and their communities.”
“Patients with CIDP face a number of diagnostic
and treatment challenges” said Jeffrey Allen, M.D., Professor,
Department of Neurology, University of Minnesota and Principal
Investigator in the ADHERE trial. “The results of the ADHERE trial
show that VYVGART Hytrulo reduces the risk of clinical
deterioration in patients with CIDP while minimizing side effects
and reducing the treatment burden. These findings enhance our
understanding of the role that IgG autoantibodies are likely to
play in the disease, and open the door to new safe, effective and
well-tolerated treatments that eliminate pathogenic IgGs.”
ADHERE Plenary Session (PL5)
Highlights Rapid, Deep
and Clinically Meaningful, and Durable
Functional Improvements in
CIDP
In the ADHERE study, a majority of patients
treated with VYVGART Hytrulo, regardless of prior treatment,
demonstrated evidence of rapid clinical improvement, and a reduced
risk of relapse compared to those treated with placebo. As
previously reported, ADHERE met its primary endpoint (p=0.000039)
demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the
risk of relapse versus placebo. VYVGART Hytrulo was well-tolerated,
and the observed safety and tolerability profile was consistent
with previous clinical trials.
- Evidence of rapid onset and
maintenance of clinical response: In the open-label Stage
A of the ADHERE study, 67% of patients treated with VYVGART Hytrulo
demonstrated evidence of clinical improvement (ECI), including 40%
who had achieved ECI by the earliest possible measure at week 4. In
Stage B, VYVGART Hytrulo-treated patients maintained a clinical
response to treatment longer than those on placebo as evidenced by
a statistically significant and clinically relevant reduction in
risk of relapse. Results across both Stage A and B indicate IgG
autoantibodies play a significant role in mediating the underlying
biology of CIDP.
- Deep and clinically
meaningful functional improvements: 81% of VYVGART
Hytrulo-treated patients demonstrated ≥1 point improvement on the
aINCAT as compared to baseline Stage A scores in ADHERE, which
includes 42% of patients with ≥2 point improvement, 28% with ≥3
point improvement, and 12% with ≥4 point improvement.
- Clinical benefit
demonstrated regardless of prior CIDP treatment: Clinical
benefit was seen across all patient subtypes, including those who
had previously received corticosteroids, intravenous or
subcutaneous immunoglobulin, or were on no treatment prior to study
entry.
- High rate of treatment
continuation: 99% of eligible patients continued to the
ADHERE-Plus open-label extension study.
- FDA decision on CIDP sBLA
expected by June 21, 2024: Data from the ADHERE trial were
submitted to the U.S. Food and Drug Administration (FDA) as part of
a supplemental Biologics License Application (sBLA) for VYVGART
Hytrulo for the treatment of CIDP. The application was accepted for
Priority Review in February 2024 and has been granted a PDUFA
target action date of June 21, 2024.
AAN presentations highlight rapid, deep,
and sustained improvements in gMG with ability to reduce steroid
burden
Clinical trial data and real-world evidence
presented during AAN continue to highlight the differentiated
efficacy and safety profile of VYVGART and VYVGART Hytrulo, driving
rapid, deep, and sustained improvement across disease scales and
with different dosing schedules, including the ability for patients
to achieve minimal symptom expression (MSE).
- MSE results in ADAPT/ADAPT+: Poster
Session 10
- ADAPT-SC+ interim results: Poster
Session 10
- ADAPT-NXT interim results: Poster
Session 10
- Cost-benefit analysis of
efgartigimod to IVIG in Canada: Poster Session 4
Side effects from long-term steroid use continue
to be a significant burden associated with autoimmune disease,
reinforcing the importance of the favorable safety profile of
VYVGART. New data presented in an oral presentation (Scientific
Platform Session 38) during AAN characterize how VVYGART treatment
can significantly reduce concomitant steroid use.
- A substantial proportion of gMG
patients (46%) were able to reduce steroid use over the first six
months of initiating VYVGART treatment, including 34% of patients
who tapered to <5mg/day and 18% who reduced completely to
zero.
- Overview of efgartigimod safety
profile across indications: Poster Session 4
- Analysis of serious infections and
malignancies in MG: Poster Session 10
About ADHERE Trial DesignThe
ADHERE trial was a multicenter, randomized, double-blind,
placebo-controlled trial evaluating VYVGART® Hytrulo (efgartigimod
alfa and hyaluronidase-qvfc) for the treatment of chronic
inflammatory demyelinating polyneuropathy (CIDP). ADHERE enrolled
322 adult patients with CIDP who were treatment naïve (not on
active treatment within the past six months or newly diagnosed) or
being treated with immunoglobulin therapy or corticosteroids. The
trial consisted of an open-label Stage A followed by a randomized,
placebo-controlled Stage B. In order to be eligible for the trial,
the diagnosis of CIDP was confirmed by an independent panel of
experts. Patients entered a run-in stage, where any ongoing CIDP
treatment was stopped and in order to be eligible for Stage A had
to demonstrate active disease, with clinically meaningful worsening
on at least one CIDP clinical assessment tool, including INCAT,
I-RODS, or mean grip strength. Treatment naïve patients were able
to skip the run-in period with proof of recent worsening. To
advance to Stage B, patients needed to demonstrate evidence of
clinical improvement (ECI) with VYVGART Hytrulo. ECI was achieved
through improvement of the INCAT score, or improvement on I-RODS or
mean grip strength if those scales had demonstrated worsening
during the run-in period. In Stage B, patients were randomized to
either VYVGART Hytrulo or placebo for up to 48 weeks. The primary
endpoint was measured once 88 total relapses or events were
achieved in Stage B and was based on the hazard ratio for the time
to first adjusted INCAT deterioration (i.e. relapse). After Stage
B, all patients had the option to roll-over to an open-label
extension study to receive VYVGART Hytrulo.
See Important Safety Information below, full
United States Prescribing Information for VYVGART and full
Prescribing Information for VYVGART Hytrulo for additional
information.
What is VYVGART® (efgartigimod
alfa-fcab) for intravenous (IV) infusion and what is VYVGART®
HYTRULO (efgartigimod alfa and hyaluronidase-qvfc) for subcutaneous
injection? VYVGART and VYVGART HYTRULO are both
prescription medicines, each 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).
IMPORTANT SAFETY INFORMATIONDo
not use VYVGART if you have a serious allergy to efgartigimod alfa
or any of the other ingredients in VYVGART. Do not use VYVGART
HYTRULO if you have a serious allergy to efgartigimod alfa,
hyaluronidase, or any of the other ingredients in VYVGART HYTRULO.
VYVGART and VYVGART HYTRULO can cause serious allergic reactions
and a decrease in blood pressure leading to fainting.
VYVGART and VYVGART HYTRULO may cause
serious side effects, including:
InfectionVYVGART and VYVGART
HYTRULO may increase the risk of infection. The most common
infections for efgartigimod alfa-fcab-treated patients were urinary
tract and respiratory tract infections. Signs or symptoms of an
infection may include 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.
Allergic Reactions
(hypersensitivity
reactions)VYVGART and VYVGART
HYTRULO can cause allergic reactions such as rashes, swelling under
the skin, and shortness of breath. Hives were also observed in
patients treated with VYVGART HYTRULO. Serious allergic reactions,
such as trouble breathing and decrease in blood pressure leading to
fainting have been reported with efgartigimod alfa-fcab.
Infusion-Related
ReactionsVYVGART and VYVGART HYTRULO can cause
infusion-related reactions. The most frequent symptoms and signs
reported with efgartigimod alfa-fcab were high blood pressure,
chills, shivering, and chest, abdominal, and back pain.
Tell your doctor if you have signs or symptoms
of an infection, allergic reaction, or infusion-related reaction.
These can happen while you are receiving your VYVGART or VYVGART
HYTRULO treatment or afterward. Your doctor may need to pause or
stop your treatment. Contact your doctor immediately if you have
signs or symptoms of a serious allergic reaction.
Before taking VYVGART or VYVGART
HYTRULO, tell your doctor if you:
- take any medicines, including
prescription and non-prescription medicines, supplements, or herbal
medicines,
- have received or are scheduled to
receive a vaccine (immunization), or
- have any allergies or medical
conditions, including if you are pregnant or planning to become
pregnant, or are breastfeeding.
What are the common side effects of
VYVGART and VYVGART HYTRULO? The most common side effects
in efgartigimod-alfa-fcab-treated patients were respiratory tract
infection, headache, and urinary tract infection. Additional common
side effects with VYVGART HYTRULO are injection site reactions,
including rash, redness of the skin, itching sensation, bruising,
pain, and hives.
These are not all the possible side effects of
VYVGART and VYVGART HYTRULO. 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
the full Prescribing Information
for VYVGART HYTRULO.
About Generalized Myasthenia
GravisGeneralized myasthenia gravis (gMG) is a rare and
chronic autoimmune disease where IgG autoantibodies disrupt
communication between nerves and muscles, causing debilitating and
potentially life-threatening muscle weakness. Approximately 85% of
people with MG progress to gMG within 24 months,1 where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population.
About Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP)Chronic inflammatory demyelinating
polyneuropathy (CIDP) is a rare and serious autoimmune disease of
the peripheral nervous system. Although confirmation of disease
pathophysiology is still emerging, there is increasing evidence
that IgG antibodies play a key role in the damage to the peripheral
nerves. People with CIDP experience fatigue, muscle weakness and a
loss of feeling in their arms and legs that can get worse over time
or may come and go. These symptoms can significantly impair a
person's ability to function in their daily lives. Without
treatment, one-third of people living with CIDP will need a
wheelchair.
About VYVGART®VYVGART is a
human IgG1 antibody fragment that binds to the neonatal Fc receptor
(FcRn), resulting in the reduction of circulating IgG
autoantibodies. It is the first approved FcRn blocker in the United
States, EU, China and Canada 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).
About VYVGART® HytruloVYVGART
Hytrulo is a subcutaneous combination of efgartigimod alfa, a human
IgG1 antibody fragment marketed for intravenous use as VYVGART®,
and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s
ENHANZE® drug delivery technology to facilitate subcutaneous
injection delivery of biologics. In binding to the neonatal Fc
receptor (FcRn), VYVGART Hytrulo results in the reduction of
circulating IgG. It is the first-and-only approved FcRn blocker
administered by subcutaneous injection.
VYVGART Hytrulo is the proprietary name in the
U.S. for subcutaneous efgartigimod alfa and recombinant human
hyaluronidase PH20. It may be marketed under different proprietary
names following approval in other regions.
About argenxargenx 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 approved neonatal Fc
receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK,
Canada and China. 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.
Contacts
Media: Ben Petokbpetok@argenx.com
Investors: Alexandra Roy
(US) aroy@argenx.com
Lynn Elton (EU) lelton@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 “aims,”
“committed,” “expects,” “may,” “will,” “potential,” "likely," or
and include statements argenx makes concerning the potential impact
of VYVGART and VYVGART Hytrulo for CIDP patients; its pioneering
approach to transforming autoimmunity redefining what is possible
for patients and their communities; the role IgG autoantibodies are
likely to play in the disease, and new safe, effective and
well-tolerated treatments that eliminate pathogenic IgGs; and the
expected FDA's decision of CIDP sBLA. 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, including but
not limited to, the results of argenx’s clinical trials,
expectations regarding the inherent uncertainties associated with
development of novel drug therapies, preclinical and clinical trial
and product development activities and regulatory approval
requirements, the acceptance of argenx's products and product
candidates by its patients as safe, effective and cost-effective,
the impact of governmental laws and regulations on its business,
and the results of its PDUFA review. 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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