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European Phase 3 Trial of Aimmune Therapeutics’ AR101 Meets Primary Endpoint

— Company Intends to Submit European MAA in Mid-2019 —

— Webcast and Conference Call Today at 8:30 a.m. EDT —

BRISBANE, Calif.–(BUSINESS WIRE)–Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical company
developing treatments for life-threatening food allergies, today
announced that its phase 3 European clinical trial of AR101 for the
treatment of peanut allergy, known as ARTEMIS (AR101 Trial
in Europe Measuring oral Immunotherapy Success),
met its primary efficacy endpoint. Topline data show that the proportion
of AR101-treated patients who tolerated a 1,000-mg dose of peanut
protein (2,043 mg cumulative) in a blinded exit challenge after
approximately nine months of AR101 treatment was significantly higher
(p<0.00001) than in the placebo group. Specifically, the median
tolerated dose of peanut protein for AR101-treated patients improved
100-fold, from 10 mg at baseline to 1,000 mg at exit. The trial also
greatly exceeded a 15% lower-bound of the 95% confidence interval (CI)
of the difference between treatment arms for all endpoints.

In addition, the safety profile and completion rate observed in ARTEMIS
are consistent with the results seen in previous AR101 clinical trials.
Notably, no cases of anaphylaxis or of eosinophilic esophagitis (EoE)
were observed. Aimmune plans to present full results in an oral
presentation at the European Academy of Allergy and Clinical Immunology
(EAACI) Congress in early June.

“We are very pleased with the results of the ARTEMIS trial, which
demonstrate that AR101 significantly improved the ability of patients to
tolerate the 1,000-mg dose of peanut protein in the exit food challenge,
which correlates to at least three or four peanuts. This level of
protection provides ample buffer beyond a typical bite of a
peanut-containing food in the real world,” said Jayson Dallas, M.D.,
President and CEO of Aimmune. “AR101 has the potential to become the
first approved therapy for peanut allergy in both the United States and
Europe, where up to two percent of children in many countries are
affected. If approved, AR101 could significantly reduce their risk of
severe, potentially life-threatening reactions to peanut exposures and
provide peace of mind to them and their families.”

The ARTEMIS findings reinforce the results from the highest level tested
in Aimmune’s landmark phase 3 PALISADE trial, which found that 50.3% of
AR101-treated patients tolerated a single highest dose of 1,000 mg of
peanut protein (2,043 mg cumulative) after approximately six months of
dose escalation followed by six months at a daily therapeutic dose of
300 mg, compared to 2.4% of placebo patients (p<0.00001). Full results
from the PALISADE trial were published in November 2018 in the New
England Journal of Medicine
.

“The ARTEMIS data demonstrate that most patients exceeded what we
consider to be protective levels well before a full year of treatment.
It’s gratifying to see how these data build upon the insights gained
throughout the entire AR101 program regarding the desensitization
process and the ability of our patients to tolerate relatively large
challenge-doses of peanut protein,” said Daniel Adelman, M.D., Chief
Medical Officer of Aimmune. “When added to our prior experiences with
AR101, these data enable us to better define the time course of the
desensitization and on-going immunomodulation processes as treatment
continues.”

The randomized, double-blind, placebo-controlled phase 3 ARTEMIS
clinical trial enrolled 175 children and adolescents ages 4 to 17 from
18 sites in France, Germany, Ireland, Italy, Spain, Sweden and the
United Kingdom. Patients underwent approximately six months of dose
escalation and then three months at a daily therapeutic dose of AR101 at
300 mg or placebo, followed by an exit double-blind, placebo-controlled
food challenge. The primary efficacy endpoint was patients’ ability to
tolerate a 1,000-mg single dose of peanut protein, the equivalent of
approximately three to four peanut kernels (2,043 mg cumulative,
equivalent to seven or eight peanut kernels).

Based on these positive results, Aimmune intends to submit a marketing
authorization application (MAA) for AR101 to the European Medicines
Agency (EMA) in mid-2019. Aimmune submitted a biologics license
application (BLA) for AR101 to the U.S. Food and Drug Administration
(FDA) in December 2018, and its review by the agency began following the
end of the U.S. government shutdown in January 2019. The FDA informed
Aimmune that the BLA will be reviewed under a 12-month target review
period, as measured from the January 2019 start date. Consequently,
review of the BLA may take until late January 2020.

Conference Call

In connection with this announcement, Aimmune Therapeutics will host a
conference call and webcast today at 8:30 a.m. EDT. To access the live
call by phone, dial (877) 497-1438 (domestic) or +1 (262) 558-6296
(international) and enter the passcode 5157603. To access a live or
recorded webcast of the call, please visit the Investor Relations
section of the Aimmune Therapeutics website at www.aimmune.com.
The recorded webcast will be available for approximately 30 days
following the call.

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 accidental 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 risk of anaphylaxis
following accidental exposures to peanut. The BLA for AR101 is under
review by the 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; Aimmune’s expectations regarding the review period of
the BLA for AR101; 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; Aimmune’s plans to present ARTEMIS results at an upcoming
conference in Europe; 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: 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; 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 Annual Report on Form 10-K for the year ended December 31, 2018. 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 have been approved for marketing by the FDA or the 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.

Contacts

Investors:
Eric Bjerkholt
(650) 376-5582 or
ebjerkholt@aimmune.com

Media:
Alison Marquiss
(650) 376-5583 or
amarquiss@aimmune.com

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