Dupixent® (dupilumab) Phase 3 trial shows positive results
in children 1 to 11 years of age with eosinophilic esophagitis
- First and only investigational
Phase 3 trial to show positive results in these young children;
results follow recent approval of Dupixent in people with
eosinophilic esophagitis aged 12 years and older who weigh at least
40 kilograms
- Trial met its primary endpoint,
with 68% of patients on higher dose Dupixent and 58% on lower dose
Dupixent achieving histological disease remission at 16 weeks
- Of the approximately 21,000
children under the age of 12 in the U.S. currently being treated
for EoE, about 9,000 do not satisfactorily respond to the
unapproved therapies they have been treated with
- Fifth pediatric pivotal trial
across three type 2 inflammatory diseases to reinforce the
well-established efficacy and safety profile of Dupixent
Paris and Tarrytown,
N.Y. July
14, 2022 A Phase
3 trial assessing the investigational use of Dupixent® (dupilumab)
in children aged 1 to 11 years with eosinophilic esophagitis (EoE)
met its primary endpoint of histological disease remission at 16
weeks with both higher and lower dose weight-tiered regimens. There
are no approved treatments for children with EoE under 12 years of
age.
Naimish Patel, M.D.Senior Vice
President, Head of Global Development, Immunology and Inflammation,
Sanofi “We are incredibly excited to share results from this Phase
3 pivotal trial evaluating Dupixent in young children suffering
from eosinophilic esophagitis – the first ever to show positive
results across a variety of primary and secondary endpoints. The
lack of treatment options for children living with eosinophilic
esophagitis leaves many caregivers with the stress and burden of
adapting their child’s meals and their entire family’s daily
schedules to ensure healthy growth and development. In some cases,
they must resort to off-label use of poorly studied treatments like
steroids that can pose serious health risks when used long term.
The faster and larger than anticipated enrollment in this trial
further emphasizes the unmet treatment needs for children with EoE
and underscores the significance of these first-ever positive
results.”
EoE is a chronic inflammatory disease that
damages the esophagus and prevents it from working properly. The
results seen with Dupixent in adults and children with EoE
demonstrate that IL-4 and IL-13 are key drivers of the type 2
inflammation underlying this disease. In children, common symptoms
of eosinophilic esophagitis include acid reflux, vomiting,
abdominal discomfort, trouble swallowing, and a failure to thrive.
These symptoms can impact growth and development, and can cause
food-related fear and anxiety which can persist through adulthood.
Diet adjustments, which oftentimes include the elimination of many
foods, is the standard treatment for EoE, as well as the use of
treatments not approved for the disease. These include proton pump
inhibitors, swallowed topical corticosteroids, or in severe cases,
a feeding tube, which may be used to ensure proper caloric intake
and weight gain. Of the approximately 21,000 children under the age
of 12 in the U.S. currently being treated for EoE, about 9,000 do
not satisfactorily respond to the unapproved therapies they have
been treated with and potentially require advanced therapy.
George D. Yancopoulos, M.D.,
Ph.D.President and Chief Scientific
Officer, Regeneron“Dupixent is the first medicine to alleviate key
signs of eosinophilic esophagitis in children as young as 1 year of
age in a Phase 3 trial. The efficacy of Dupixent demonstrates that,
in this age group, as in adults, IL-4 and IL-13 are key drivers of
the type 2 inflammation underlying this debilitating disease.
Eosinophilic esophagitis can turn the basic and life-sustaining act
of eating into a painful experience at a point in children’s lives
when proper nutrition and achieving a healthy weight is critical to
ensuring they grow and thrive. The positive results from this Phase
3 pediatric trial show Dupixent has the potential to improve signs
of eosinophilic esophagitis and support healthy weight gain in
children from their first birthday.”
In the Phase 3 trial, 102 children aged 1 to 11
were randomized to receive Dupixent, in either a higher dose (n=37)
or lower dose (n=31) regimen based on body weight, or placebo
(n=34). At 16 weeks, 68% of children on higher dose and 58% of
patients on lower dose Dupixent achieved the primary endpoint of
significant histological disease remission (peak esophageal
intraepithelial eosinophil count of ≤6 eosinophils [eos]/high power
field [hpf]) compared to 3% of children on placebo (p<0.0001 for
both). Additionally, children receiving higher dose Dupixent
experienced the following changes at week 16:
- 86% reduction in peak esophageal
intraepithelial eosinophil count from baseline compared to a 21%
increase for placebo (p<0.0001).
- 0.88 and 0.84 reduction from
baseline in disease severity and extent, respectively, as measured
at the microscopic level in biopsy specimens compared to a 0.02 and
0.05 increase for placebo (both p<0.0001).
- 3.5-point reduction in abnormal
endoscopic findings from baseline compared to a 0.3-point increase
for placebo (p<0.0001).
- A numerical improvement in the
proportion of days children experienced symptoms of EoE from
baseline, as reported by caregivers (Pediatric EOE signs/symptoms
questionnaire [PESQ-C]), compared to placebo, though not
statistically significant. The PESQ-C is a novel endpoint developed
by Sanofi and Regeneron used for the first time in this trial,
designed to assess symptoms in young children through their
caregivers (as signs), as children may have difficulty verbalizing
their symptoms themselves.
- As part of a prespecified
exploratory analysis a 3.09 percentile increase in body weight for
age percentile from baseline compared to 0.29 for placebo.
Histological, anatomic and cellular secondary
endpoints were also analyzed for the lower dose group, with all
being nominally significant and generally comparable with the
higher dose. More detailed results will be shared at an upcoming
medical meeting, including additional data for the endpoints in the
lower dose group.
Safety results were generally consistent with
the known safety profile of Dupixent in its approved EoE indication
for children and adults aged 12 years and older who weigh at least
40 kg. For the 16-week treatment period, overall rates of adverse
events (AEs) were 79% for Dupixent and 91% for placebo. AEs more
commonly (≥5%) observed with Dupixent compared to placebo included
COVID-19 (21% Dupixent, 0% placebo; all cases were mild or moderate
and did not lead to study discontinuation), rash (9% Dupixent, 6%
placebo), headache (8% Dupixent, 3% placebo), viral gastroenteritis
(6% Dupixent, 3% placebo), diarrhea (6% Dupixent, 3% placebo) and
nausea (6% Dupixent, 0% placebo). Rates of treatment
discontinuation due to AEs prior to week 16 were 0% for Dupixent
and 6% for placebo.
In May 2022, the U.S. Food and Drug
Administration (FDA) approved Dupixent 300 mg weekly to treat
patients with EoE aged 12 years and older and weighing at least 40
kg after granting the medicine Priority Review.
The potential use of Dupixent in children with
EoE aged 1 to 11 years is currently under clinical development, and
the safety and efficacy have not been fully evaluated by any
regulatory authority. These data will be discussed with regulatory
authorities around the world, starting with the U.S. later this
year.
About the
Dupixent Pediatric
Eosinophilic Esophagitis
Trial
The Phase 3, randomized, double-blind,
placebo-controlled trial evaluated the efficacy and safety of
Dupixent in young children aged 1 to 11 years with EoE, as
determined by histological and patient- or caregiver-reported
measures. At baseline, 98% of these patients had at least one
co-existing type 2 inflammatory disease such as food allergy,
allergic rhinitis, asthma and atopic dermatitis.
Patients received Dupixent subcutaneously at
either a higher dose or lower dose regimen based on their weight
(ranging from ≥5 kg to <60 kg) over a 16-week period, at which
point all endpoints were assessed. The dosing frequency ranged
between every two weeks and every four weeks, based on weight.
The primary endpoint was histological disease
remission. Secondary endpoints included histopathologic measures of
the severity and extent of tissue scarring in the esophagus
(EoE-HSS grade and stage scores, which measure changes in eight
cellular and tissue features on 0-3 scales, respectively), and
abnormal endoscopic findings (EoE Endoscopic Reference Score
[EoE-EREFS] on a 0-18 scale), as well as changes in
caregiver-reported symptoms (proportion of days with 1 or more EoE
signs [e.g. stomach pain, vomiting, food refusal] by the Pediatric
EoE Sign/Symptom Questionnaire-caregiver version [PESQ-C]). An
exploratory endpoint assessed change from baseline in body weight
for age percentile.
The trial is ongoing with a 36-week extended
active treatment period to evaluate long-term outcomes.
About Dupixent
Dupixent is a fully human monoclonal antibody
that inhibits the signaling of the interleukin-4 (IL-4) and
interleukin-13 (IL-13) pathways and is not an immunosuppressant.
The Dupixent development program has shown significant clinical
benefit and a decrease in type 2 inflammation in Phase 3 trials,
establishing that IL-4 and IL-13 are key and central drivers of the
type 2 inflammation that plays a major role in multiple related and
often co-morbid diseases. These diseases include approved
indications for Dupixent such as asthma, atopic dermatitis, chronic
rhinosinusitis with nasal polyposis (CRSwNP) and EoE, as well as
investigational diseases such as prurigo nodularis.
Dupixent has received regulatory approvals
around the world for use in certain patients with atopic
dermatitis, asthma, CRSwNP or EoE in different age populations.
Dupixent is currently approved across these indications in the U.S.
and for one or more of these indications in more than 60 countries,
including in the European Union and Japan. More than 400,000
patients have been treated with Dupixent globally.
Dupilumab Development
Program
Dupilumab is being jointly developed by Sanofi
and Regeneron under a global collaboration agreement. To date,
dupilumab has been studied across more than 60 clinical trials
involving more than 10,000 patients with various chronic diseases
driven in part by type 2 inflammation.
In addition to the currently approved
indications, Sanofi and Regeneron are studying dupilumab in a broad
range of diseases driven by type 2 inflammation or other allergic
processes in Phase 3 trials, including prurigo nodularis, pediatric
EoE, hand and foot atopic dermatitis, chronic inducible
urticaria-cold, chronic spontaneous urticaria, chronic pruritis of
unknown origin, chronic obstructive pulmonary disease with evidence
of type 2 inflammation, chronic rhinosinusitis without nasal
polyposis, allergic fungal rhinosinusitis, allergic
bronchopulmonary aspergillosis and bullous pemphigoid. These
potential uses of dupilumab are currently under clinical
investigation, and the safety and efficacy in these conditions have
not been fully evaluated by any regulatory authority.
About Regeneron
Regeneron is a leading biotechnology company
that invents, develops and commercializes life-transforming
medicines for people with serious diseases. Founded and led for
nearly 35 years by physician-scientists, our unique ability to
repeatedly and consistently translate science into medicine has led
to numerous FDA-approved treatments and product candidates in
development, almost all of which were homegrown in our
laboratories. Our medicines and pipeline are designed to help
patients with eye diseases, allergic and inflammatory diseases,
cancer, cardiovascular and metabolic diseases, pain, hematologic
conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the
traditional drug development process through our proprietary
VelociSuite® technologies, such as VelocImmune®, which uses unique
genetically humanized mice to produce optimized fully human
antibodies and bispecific antibodies, and through ambitious
research initiatives such as the Regeneron Genetics Center, which
is conducting one of the largest genetics sequencing efforts in the
world.
For more information, please visit
www.Regeneron.com or follow @Regeneron on Twitter.
About SanofiWe are an innovative global
healthcare company, driven by one purpose: we chase the miracles of
science to improve people’s lives. Our team, across some 100
countries, is dedicated to transforming the practice of medicine by
working to turn the impossible into the possible. We provide
potentially life-changing treatment options and life-saving vaccine
protection to millions of people globally, while putting
sustainability and social responsibility at the center of our
ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ:
SNY.
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