–APPEAL 2: Struggle to Avoid Accidental Peanut Exposure Negatively
Impacts Quality of Life for Patients and Families–
–Phase 3 PALISADE Follow-on Study Analysis: Improvements in Quality
of Life for Peanut-Allergic Patients after Continued AR101 Treatment–
LISBON, Portugal–(BUSINESS WIRE)–Aimmune Therapeutics, Inc. (Nasdaq:AIMT), a biopharmaceutical company
developing treatments for life-threatening food allergies, today
announced new data from two studies that assessed the psychosocial
burden of living with peanut allergy. Results from APPEAL 2, the first
European multi-country qualitative evaluation of the impact of living
with peanut allergy, found that living in fear of a potentially fatal
reaction to peanuts significantly impacts the quality of life of
individuals with peanut allergy and their families. In addition, data
from an analysis of patients who received daily treatment with AR101
during the open-label extension of the Phase 3 PALISADE trial, ARC004,
showed they experienced clinically meaningful improvements in
disease-specific quality of life. AR101 is an investigational biologic
drug for use in oral immunotherapy as a treatment to reduce the
frequency and severity of allergic reactions following exposure to
peanuts. These data were presented here in a late-breaking poster
discussion session and in an oral session, respectively, at the European
Academy of Allergy and Clinical Immunology (EAACI) Congress 2019 in
Lisbon.
“Across Europe, individuals and families are failing to cope with peanut
allergy through a strategy of avoidance of peanuts,” said Audrey
DunnGalvin, Ph.D., investigator of both APPEAL 1 and APPEAL 2 and a
lecturer in the School of Applied Psychology at University College Cork
in Cork, Ireland. “Both our qualitative and quantitative research shows
how fear, frustration and uncertainty are the hallmarks of living with
peanut allergy, negatively affecting the day-to-day lives of both
patients and their families.”
APPEAL 2 was conducted to gather qualitative data to build upon the
findings of APPEAL 1, the first European multi-country quantitative
study to assess the psychosocial burden and impact on daily activities
in peanut-allergic individuals and their families.
The results of APPEAL 2 amplify the findings of APPEAL 1, showing that
peanut allergy affects the lives of peanut-allergic individuals both
emotionally and socially, as well as their relationships and daily
coping behaviors as they attempt to avoid peanuts and prepare for a
reaction in the event of an accidental exposure. Furthermore,
peanut-allergic individuals are more likely to experience feeling
different, isolated, and restricted of social activities as compared
with caregivers, who more often experience stress and adverse impacts on
work and career. Both those with peanut allergy and caregivers
experience anxiety, worry, sadness, and annoyance, and reported their
lives are adversely affected by peanut avoidance, reactions to peanuts,
and the fear of reacting to peanuts.
Separately, data from the six-month open-label extension study of
PALISADE showed that children receiving the therapeutic dose of AR101
(300 mg/day) for an additional 28 weeks reported improvements in all
domains of the Food Allergy Quality of Life Questionnaire (FAQLQ), as
well as in the self-reported food allergy specific independent measure
(FAIM). The mean (95% CI) change in self-reported FAQLQ domains was
greater than the estimated minimal important difference (MID), or the
smallest benefit that a patient would say is valuable. Furthermore,
improvements over time in domains of FAQLQ and FAIM may reflect the need
for the patients and caregivers to adjust to their desensitization
status. These improvements in quality of life are consistent with recent
findings from a two-year longitudinal study of changes in quality of
life due to peanut oral immunotherapy and represent the first data from
a clinical trial showing positive changes in food allergy-related
quality of life.
“We are encouraged that daily dosing with AR101 in the open-label
extension trial resulted in important and clinically meaningful
improvements in disease-specific quality-of-life measures in children
with peanut allergy,” said Prof. Jonathan Hourihane, MB, BCh., BAO,
investigator with the PALISADE trial and Professor of Paediatrics and
Child Health at University College Cork in Ireland. “Given that quality
of life improves in treated patients once they know they have been
desensitized, treatment with AR101 may help relieve the anxiety, fear,
and social limitations that are a current reality for individuals and
families who live with peanut allergy.”
About the APPEAL Studies
APPEAL 1 (Allergy to Peanuts ImPacting Emotions
And Life 1) collected data from 1,846 participants in
eight European countries and was the first pan-European quantitative,
cross-sectional survey that explored the psychosocial impacts of living
with PA with use of a novel questionnaire. APPEAL 2 (Allergy to Peanuts
ImPacting Emotions And Life 2), was the
first qualitative evaluation of the impact of living with peanut allergy
conducted across multiple European countries. Researchers interviewed
146 individuals with moderate-to-severe peanut allergies– 39 adults ages
18 to 30 years, 39 adolescents ages 13 to 17 years, and 24 children ages
8 to 12 years, as well as 44 caregivers of peanut-allergic individuals
ages 4 to 17 years. The goal of the study was to identify concepts
important to peanut-allergic individuals and their parents/caregivers,
as well as to compare and contrast the impact of peanut allergy between
countries and groups.
About the PALISADE Open-Label Extension Trial
A total of 110 children ages 4 to 17 with peanut allergy participated in
a six-month open-label extension trial of daily AR101 maintenance
therapy after they had completed the double-blind, placebo-controlled,
Phase 3 PALISADE trial. Of these, 68 children and 93 parents/caregivers
completed an age-appropriate Food Allergy Quality of Life Questionnaire
(FAQLQ) at screening and after the open-label extension. At screening,
67.3% of participants had experienced one or more anaphylactic reactions
to peanut allergy in their lifetime.
About PALISADE
The international, randomized, double-blind, placebo-controlled Phase 3
PALISADE (Peanut Allergy oral Immunotherapy Study
of AR101 for Desensitization) trial evaluated the efficacy
and safety of AR101 in patients with peanut allergy. PALISADE was
conducted at 66 sites in 10 countries in North America and Europe. A
total of 496 patients ages 4 to 17 were randomized 3:1 to receive AR101
or placebo along with 55 adults ages 18 to 49 who were not part of the
primary analysis. To meet PALISADE’s inclusion criteria, patients could
tolerate no more than the 30-mg dose of peanut protein in an entry
double-blind, placebo-controlled food challenge (DBPCFC), which
consisted of consecutive doses of 1, 3, 10, 30 and 100 mg of peanut
protein, given 20 to 30 minutes apart, and associated with only mild
symptoms.
Patients enrolled in PALISADE underwent a dose escalation period of
approximately 22 weeks to reach a therapeutic dose of 300 mg per day of
AR101 or placebo, then continued with the daily therapeutic dose at 300
mg per day of AR101 or placebo for approximately six months. At that
point, patients underwent an exit DBPCFC, which tested consecutive doses
of 3, 10, 30, 100, 300, 600 and 1,000 mg of peanut protein, given 20 to
30 minutes apart, and associated with only mild symptoms. Both the entry
and exit DBPCFCs used an independent, blinded assessor. Following the
completion of the exit DBPCFC, patients were unblinded and eligible to
rollover or crossover into the follow-on ARC004 clinical trial, as
appropriate.
Full results from the PALISADE trial were published in the New
England Journal of Medicine in November 2018.1
About Peanut Allergy
Peanut allergy is one of the most common food allergies, affecting more
than 6 million people in the U.S. and Europe, and reactions to peanut
are often severe and potentially life-threatening. Peanut allergy
usually persists into adulthood2,3,4, 5 and while rare,
accounts for the majority of deaths related to food allergy.6
There are no approved treatment options for peanut allergy.7
The standard of care has been a strict elimination diet and the timely
administration of rescue medications in case of an allergic reaction
from accidental exposure.8,9,10 Despite vigilance, accidental
exposures may occur11 and cause reactions of unpredictable
severity,12 leading to a lifelong risk of severe reactions.
About AR101
AR101 is a new, peanut-derived investigational oral biologic drug for
use in oral immunotherapy in patients with peanut allergy. The drug,
which is manufactured in accordance with current Good Manufacturing
Practices (cGMP), delivers a daily dose of peanut protein with a
consistent protein profile, analyzed to ensure reliable major allergen
content. The amount of active ingredient in each AR101 capsule is
controlled to ensure minimal variability of allergen content across
doses of a given strength. AR101 is administered as an oral powder in
graduated doses in pull-apart capsules or foil-laminate sachets. The
contents are mixed thoroughly with a few spoonfuls of age-appropriate,
unheated food of the patient’s choice.
Aimmune’s Biologics License Application (BLA) for AR101 was accepted for
review by the U.S. Food and Drug Administration (FDA) in March 2019. The
Allergenic Products Advisory Committee (APAC) of the FDA will review the
BLA for AR101 at its meeting scheduled for September 13, 2019. The
company plans to submit a Marketing Authorization Application (MAA) for
AR101 to the European Medicines Agency in mid-2019.
About Aimmune Therapeutics
Aimmune Therapeutics, Inc., is a biopharmaceutical company developing
oral treatments for life-threatening food allergies. The company’s Characterized
Oral Desensitization
ImmunoTherapy
(CODIT™) approach is intended to provide meaningful levels of protection
against allergic reactions resulting from exposure to food allergens by
desensitizing patients with defined, precise amounts of key allergens.
Aimmune’s first investigational biologic product, AR101, is being
developed as a treatment to reduce the frequency and severity of adverse
events following exposure to peanut. The BLA for AR101 is under review
by the U.S. FDA, which in 2015 granted AR101 Breakthrough Therapy
Designation for the desensitization of peanut-allergic patients 4 to 17
years of age. Aimmune expects to file for marketing approval of AR101 in
Europe in mid-2019. Aimmune has filed an IND application for its second
product, AR201 for the treatment of egg allergy, and intends to start a
randomized Phase 2 clinical trial in mid-2019. For more information,
please see www.aimmune.com.
Forward-Looking Statements
Statements contained in this press release regarding matters that are
not historical facts are “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995. Because such
statements are subject to risks and uncertainties, actual results may
differ materially from those expressed or implied by such
forward-looking statements. Such statements include, but are not limited
to, statements regarding: Aimmune’s expectations regarding the potential
benefits of AR101, including relief from emotional and other factors
that negatively impact quality of life; Aimmune’s expectations regarding
the review of the BLA for AR101 by the FDA and APAC; Aimmune’s
expectations regarding the planned timing and filing for marketing
approval of AR101 in Europe; Aimmune’s expectations on the timing of
initiating a phase 2 clinical trial for AR201;and Aimmune’s expectations
regarding potential applications of the CODIT™ approach to treating
life-threatening food allergies. Risks and uncertainties that contribute
to the uncertain nature of the forward-looking statements include:
Aimmune’s or any of its collaborative partners’ ability to initiate
and/or complete clinical trials; the unpredictability of the regulatory
process; the possibility that Aimmune’s or any of its collaborative
partners’ clinical trials will not be successful; Aimmune’s dependence
on the success of AR101; Aimmune’s reliance on third parties for the
manufacture of Aimmune’s product candidates; possible regulatory
developments in the United States and foreign countries; and Aimmune’s
ability to attract and retain senior management personnel. These and
other risks and uncertainties are described more fully in Aimmune’s most
recent filings with the Securities and Exchange Commission, including
its Quarterly Report on Form 10-Q for the quarter ended March 31, 2019.
All forward-looking statements contained in this press release speak
only as of the date on which they were made. Aimmune undertakes no
obligation to update such statements to reflect events that occur or
circumstances that exist after the date on which they were made.
This press release concerns AR101, a product candidate that is under
clinical investigation, and AR201, a product candidate that Aimmune
expects will be under clinical investigation in 2019. Neither AR101 nor
AR201 has been approved for marketing by the FDA or the European
Medicines Agency (EMA). AR101 and AR201 are currently limited to
investigational use, and no representation is made as to their safety or
effectiveness for the purposes for which they are being investigated.
# # #
References
1 Vickery BP, Vereda A, Casale TB, et al. AR101 oral
immunotherapy for peanut allergy. New Engl J Med. 2018; DOI:
10.1056/NEJMoa1812856.
2 Crespo JF, James JM,
Fernandez-Rodriguez C, Rodriguez J. Food allergy: nuts and tree nuts. Br
J Nutr. 2006; 96:Suppl 2:S95-S102.
3 Moreno MA.
Guidelines for children with peanut allergy. JAMA Pediatr.
2017;171:100.
4 Skolnick HS, Conover-Walker MK, Koerner
CB, Sampson HA, Burks W, Wood RA. The natural history of peanut allergy. J
Allergy Clin Immunol. 2001;107:367-74.
5 Fleischer
DM, Conover-Walker MK, Christie L, Burks AW, Wood RA. The natural
progression of peanut allergy: resolution and the possibility of
recurrence. J Allergy Clin Immunol. 2003;112:183-9.
6
Bock SA, Muñoz-Furlong A, Sampson HA. Fatalities due to anaphylactic
reactions to foods. J Allergy Clin Immunol. 2001;107:191-3.
7
Yu W, Freeland DMH, Nadeau KC. Food allergy: immune mechanisms,
diagnosis and immunotherapy. Nat Rev Immunol. 2016;16:751-65.
8
Boyce JA, Assa’ad A, Burks AW, et al. Guidelines for the diagnosis and
management of food allergy in the United States: report of the
NIAID-sponsored expert panel. J Allergy Clin Immunol.
2010;126:Suppl:S1-S58.
9 Sampson HA, Aceves S, Bock SA,
et al. Food allergy: a practice parameter update — 2014. J Allergy
Clin Immunol. 2014;134(5):1016-25.e43.
10 Muraro A,
Werfel T, Hoffmann-Sommergruber K, et al. EAACI food allergy and
anaphylaxis guidelines: diagnosis and management of food allergy. Allergy.
2014;69:1008-25.
11 Rimbaud L, Heraud F, La Vieille S,
Leblanc J-C, Crépet A. Quantitative risk assessment relating to the
inadvertent presence of peanut allergens in various food product. Int
Food Risk Anal J. 2013;3:1-11.
12 Allen KJ,
Remington BC, Baumert JL, et al. Allergen reference doses for
precautionary labeling (VITAL 2.0): clinical implications. J Allergy
Clin Immunol. 2014;133:156-64.
Contacts
Investors:
Eric Bjerkholt
+1-(650) 376-5582 or
ebjerkholt@aimmune.com
Media:
Jerica
Pitts
+1-(312) 858-3469
jpitts@w2ogroup.com
Louise
Strong
+44 203 808 6471
lstrong@w2ogroup.com