Aimmune Therapeutics, Inc. (Nasdaq: AIMT), a biopharmaceutical
company developing treatments for life-threatening food allergies,
today announced financial results for the quarter and nine months
ended September 30, 2017. As of September 30, 2017, cash, cash
equivalents, and investments totaled $212.0 million.
Aimmune also announced that CEO Stephen Dilly, M.B.B.S., Ph.D.,
plans to retire by the end of 2018. Aimmune will initiate a search
for a successor CEO to lead the Company as it builds towards the
potential commercial launch of its lead investigational product,
AR101, which is currently in Phase 3 development for the treatment
of peanut allergy. Dr. Dilly will continue as Aimmune’s CEO until
his replacement joins the company and will be available through a
transition period.
“My decision to retire is based solely on my personal desire to
have more time for my family, especially my eldest son, who has
special needs,” said Dr. Dilly. “We are announcing this now in
order to facilitate an orderly executive search and transition
period, and I remain completely committed to continuing to lead
Aimmune through the exciting events ahead. We are looking forward
to the completion of our pivotal Phase 3 PALISADE trial around
year-end and sharing top-line data in the first quarter of
2018.”
“We continue to execute well on all fronts. In the third
quarter, we were very pleased to announce a Phase 2 clinical
collaboration with Regeneron and Sanofi that will explore the
potential of AR101 and adjunctive dupilumab to achieve sustained
unresponsiveness to peanut,” continued Dr. Dilly. “In addition, we
are making solid progress in RAMSES, ARTEMIS, and ARC004, three
additional trials that will support our regulatory submissions for
AR101 at the end of 2018. We are also on track to file an IND for
our egg CODIT program in 2018. Financially, we continue to be in a
strong position to support our planned development activities
through regulatory submissions of AR101.”
“Based on what Aimmune has accomplished, the board and
management are grateful for the tremendous contributions Stephen
has made. We respect his very personal decision and appreciate the
fact that his deep and sustained commitment allows us to spearhead
a fulsome search for the right next CEO of Aimmune, an individual
with significant commercial and strategic experience, to build upon
what Stephen and the team have delivered to date. We have never
been more excited about Aimmune’s potential, and will work
deliberately to identify and land our next leader while the team
led by Stephen maintains focus on our 2017 and 2018 objectives,”
added Mark McDade, Chairman of the Board.
Recent Corporate Highlights
Announced Phase 2 Clinical Collaboration with
Regeneron/Sanofi. In October, Aimmune announced a clinical
collaboration with Regeneron and its strategic alliance
collaborator Sanofi to study AR101 treatment with adjunctive
dupilumab in peanut-allergic patients in a Phase 2 clinical trial.
Regeneron will sponsor the trial, with Aimmune to provide clinical
supply of AR101 and food challenge materials. The clinical
collaboration will include the formation of an
Aimmune–Regeneron/Sanofi Joint Development Committee.
The planned Phase 2 clinical trial is expected to begin in 2018
with a proposed primary endpoint of tolerating a certain dose of
peanut protein in a double-blind, placebo-controlled food challenge
(DBPCFC) that will include doses matching and exceeding those being
tested in current AR101 studies. The study also includes a proposed
exploration of sustained unresponsiveness after discontinuation of
therapy in another DBPCFC. Sustained unresponsiveness is achieved
when, after a break in treatment, peanut-allergic patients are able
to tolerate a defined amount of peanut protein with no more than
mild allergic symptoms.
Published ARC001 Phase 2 Results in Peer-Reviewed
Journal. In October, Aimmune announced the publication of
results from its Phase 2 ARC001 trial of AR101 in The Journal of
Allergy and Clinical Immunology: In Practice1. This was the first
peer-reviewed publication of efficacy and safety of an
industry-sponsored food allergy trial. The ARC001 study
demonstrated that approximately six months of AR101 treatment
significantly raised the level of tolerance of peanut protein,
compared to placebo.
The observed differences in response rate between AR101 and
placebo groups on the Intent-to-Treat (ITT) analysis were 60
percent for the 300-mg endpoint (95% CI 34-87%) and 62 percent for
the 600-mg endpoint (95% CI 37-87%). The lower bound of the 95%
confidence interval on the ARC001 ITT analysis greatly exceeded the
minimally clinically meaningful difference of 15 percent agreed to
with the Food and Drug Administration (FDA) for the Phase 3
PALISADE study primary endpoint. Consistent with the known
mechanisms of oral immunotherapy, transient allergic symptoms
occurred in nearly all AR101 subjects, with 96 percent of those
symptoms being mild, not dose-limiting, and not requiring medical
intervention. No adverse events were graded as severe.
Gastrointestinal symptoms were the most common treatment-related
adverse events in both the AR101 and the placebo subjects.
Announced Publication of Clinical Data Demonstrating the
Potential of AR101 to Reduce Peanut-Specific TH2
Cells. In August, Aimmune announced that AR101 was featured in
a publication by Benaroya Research Institute in the August 2 issue
of Science Translational Medicine2 focused on the discovery of an
immune cell subset — TH2A cells – that appears to be involved in
the pathogenesis of allergies. These allergen-specific T cells are
present in people with allergies but nearly entirely absent from
people without allergies.
In a small pilot experiment, AR101 treatment was associated with
a statistically significant reduction of TH2A cells in blood
samples from a subset of peanut-allergic patients from Aimmune’s
ARC001 trial. Specifically, patients receiving AR101 experienced
statistically significant reductions in TH2A cells, whereas there
was no reduction in TH2A cells in patients receiving placebo. These
TH2A cell reductions appeared to be associated with clinical
response.
Upcoming Milestones
Completion of PALISADE Around Year-End 2017; Top-Line Data in
First Quarter 2018. Aimmune expects that the last double-blind,
placebo-controlled food challenge (DBPCFC) in its Phase 3 PALISADE
trial of AR101 will be conducted in December 2017 and that top-line
data will be available in the first quarter of 2018.
There are two statistical analysis plans: one is designed to
support a Biologics License Application (BLA) with the U.S. Food
and Drug Administration (FDA), and the other to support a Marketing
Authorization Application (MAA) with the European Medicines Agency
(EMA). The primary endpoint for the BLA is the proportion of
subjects ages 4–17 who tolerate a single highest dose of at least
600 mg in the exit DBPCFC after six months of maintenance therapy.
As part of the statistical analysis plan’s requirements for success
as agreed with the FDA, the study must demonstrate at least a 15
percent superiority margin of the AR101 arm over the placebo arm;
the study is powered greater than 90 percent to detect this
difference. Tolerating single highest doses of at least 300 mg and
1,000 mg are secondary endpoints for the BLA. The primary endpoint
for the MAA is the proportion of subjects ages 4–17 who tolerate a
single highest dose of at least 1,000 mg. There is no requirement
to demonstrate a 15 percent margin of superiority for the MAA.
Tolerating single highest doses of at least 300 mg and 600 mg are
secondary endpoints for the MAA.
Third Quarter Financial Results
For the quarter and nine months ended September 30, 2017, net
loss was $31.8 million and $90.2 million, respectively, compared to
net loss of $22.1 million and $55.7 million for the comparable
periods of 2016.
On a per share basis, net loss for the quarter and nine months
ended September 30, 2017, was $0.63 and $1.79, respectively,
compared to net loss per share of $0.53 and $1.33 for the
comparable periods of 2016. The weighted average shares outstanding
for each of the quarter and nine months ended September 30, 2017
were 50.5 million and 50.3 million shares, respectively, compared
to 42.0 million and 41.8 million shares for the comparable periods
in 2016.
Research and development expenses for the quarter and nine
months ended September 30, 2017, were $21.1 million and $60.7
million, respectively, compared to $15.9 million and $37.7 million
for the comparable periods in 2016. The increase was primarily due
to the development of AR101, including progression of the PALISADE
trial, enrollment of patients in the open-label follow-on study of
PALISADE (ARC004) and the real-life RAMSES trial, and contract
manufacturing costs.
General and administrative expenses for the quarter and nine
months ended September 30, 2017, were $11.2 million and $31.0
million, respectively, compared to $6.4 million and $18.5 million
for the comparable periods in 2016. The increase was primarily due
to additional employee-related costs, including stock-based
compensation expense, and external professional services as Aimmune
continues to build the infrastructure to support the development
and potential commercialization of AR101.
Cash, cash equivalents, and investments totaled $212.0 million
at September 30, 2017, compared to $282.5 million at December 31,
2016. The decrease primarily reflects cash used in operations.
About Aimmune Therapeutics
Aimmune Therapeutics, Inc., is a clinical-stage
biopharmaceutical company developing treatments for
life-threatening food allergies. The company’s Characterized Oral
Desensitization ImmunoTherapy (CODIT™) approach is intended to
achieve meaningful levels of protection by desensitizing patients
with defined, precise amounts of key allergens. Aimmune’s first
investigational biologic product using CODIT™, AR101 for the
treatment of peanut allergy, has received the FDA’s Breakthrough
Therapy Designation for the desensitization of peanut-allergic
patients 4-17 years of age and is currently being evaluated in
Phase 3 clinical trials. For more information, please see
www.aimmune.com.
References
1. JA Bird et al. (2017) Efficacy and safety
of AR101 in oral immunotherapy for peanut allergy: results of
ARC001, a randomized, double-blind, placebo-controlled Phase 2
clinical trial. Journal of Allergy and Clinical Immunology: In
Practice.
DOI:
http://dx.doi.org/10.1016/j.jaip.2017.09.016.
2. E Wambre et al. (2017) A phenotypically
and functionally distinct human TH2 cell subpopulation is
associated with allergic disorders. Science Translational
Medicine.
DOI:
http://dx.doi.org/10.1126/scitranslmed.aam9171.
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 for its Phase 3 PALISADE trial of AR101,
including that final study visits will be completed around year-end
2017 and that topline data for the trial will be available in the
first quarter of 2018; Aimmune’s expectation that it will recruit
and hire a new chief executive officer by the end of 2018;
Aimmune’s expectation that it will file regulatory approval
applications for AR101 by the end of 2018; Aimmune’s expectations
that it will file an IND for its egg allergy program by the end of
2018; Aimmune’s expectations for its RAMSES, ARTEMIS and ARC004
trials of AR101; Aimmune’s expectations regarding the anticipated
timing of any future clinical trials, including the Phase 2
clinical trial to be sponsored by Regeneron and Sanofi;
Aimmune’s expectations regarding the potential benefits of AR101,
including in combination with dupilumab; Aimmune’s expectations
regarding the sufficiency of its capital resources; 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 expectation that Aimmune
will need additional funds to finance its operations; 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 its product candidates; and possible
regulatory developments in the United States and foreign countries.
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 September 30, 2017. 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 a product that is under clinical
investigation and that has not yet been approved for marketing by
the U.S. Food and Drug Administration (FDA) or the European
Medicines Agency (EMA). It is currently limited to investigational
use, and no representation is made as to its safety or
effectiveness for the purposes for which it is being
investigated.
AIMMUNE THERAPEUTICS, INC. CONDENSED CONSOLIDATED
BALANCE SHEETS (In thousands)
September 30,
2017
December 31,
(Unaudited)
2016 (1)
Assets Cash and cash equivalents $ 73,821 $ 124,010
Short-term investments 138,209 124,921 Prepaid expenses and other
current assets 6,063 2,749 Total current assets
218,093 251,680 Long-term investments — 33,602 Property and
equipment, net 14,940 10,391 Prepaid expenses and other assets
637 3,116 Total assets $ 233,670 $ 298,789
Liabilities and Stockholders’ Equity Current liabilities $
21,187 $ 11,450 Other liabilities 1,894 1,367 Stockholders’ equity
210,589 285,972 Total liabilities and stockholders’
equity $ 233,670 $ 298,789 (1) Derived from the audited
financial statements, included in the Company's Annual Report on
Form 10-K for the year ended December 31, 2016.
AIMMUNE THERAPEUTICS, INC. CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS (In thousands, except per share
amounts) Quarter Ended Nine
Months Ended September 30, September 30,
2017 2016 2017 2016
Operating Expenses Research and development(1) $ 21,063 $ 15,888 $
60,671 $ 37,684 General and administrative(1) 11,226
6,353 30,963 18,542 Total operating expenses
32,289 22,241 91,634 56,226 Loss from
operations (32,289 ) (22,241 ) (91,634 ) (56,226 ) Interest income,
net 497 155 1,475 478 Net loss $
(31,792 ) $ (22,086 ) $ (90,159 ) $ (55,748 ) Net loss per
common share, basic and diluted $ (0.63 ) $ (0.53 ) $ (1.79 ) $
(1.33 ) Shares used in computing net loss per common share, basic
and diluted 50,458 41,997 50,254 41,831
(1) Includes stock-based compensation
expenses of:
Quarter Ended Nine Months Ended
September 30, September 30, 2017 2016
2017 2016 Research and development $ 1,305 $ 1,493 $
3,482 $ 3,710 General and administrative 2,774 1,995
8,389 5,383 Total stock-based compensation expenses $
4,079 $ 3,488 $ 11,871 $ 9,093
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version on businesswire.com: http://www.businesswire.com/news/home/20171106005542/en/
Aimmune Therapeutics, Inc.InvestorsLaura Hansen, Ph.D.,
650-396-3814lhansen@aimmune.comorMediaAlison Marquiss,
650-376-5583amarquiss@aimmune.com
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