Aimmune Therapeutics Announces Publication in The Journal of Allergy and Clinical Immunology: In Practice of Positive AR101 Data from ARC001 Phase 2 Clinical Trial
— Study Showed that Six Months of AR101 Treatment Could Significantly Reduce Clinical Reactivity to Peanut in Approximately 80 Percent of Subjects Enrolled —
— First Peer-Reviewed Publication of Efficacy and Safety of an Industry-Sponsored Food Allergy Trial —
The ARC001 study enrolled 55 peanut-allergic subjects, ages 4–21. The study was the first randomized, placebo-controlled Phase 2 peanut oral immunotherapy study to include double-blind, placebo-controlled food challenges at both entry and exit. The study was also the first conducted using an investigational oral biologic drug with a characterized peanut protein profile, manufactured to cGMP standards and intended for potential commercialization.
The ARC001 study met its primary endpoint, providing evidence that AR101 has immunomodulatory activity, which, after approximately six months of treatment, significantly reduced clinical reactivity in almost 80 percent of the peanut-allergic subjects randomized to AR101 treatment.
"ARC001 was the first Phase 2 peanut OIT study to use sufficiently
rigorous design features, providing preliminary but high-quality
evidence that AR101 has immunomodulatory activity that can significantly
reduce clinical reactivity to peanut in an appropriately supervised
clinical setting," said
The primary endpoint of the Phase 2 study was the proportion of subjects in each arm able to tolerate a single highest dose of at least 300 mg of peanut protein, with no or only mild symptoms, at an exit double-blind, placebo-controlled food challenge (DBPCFC). The exit DBPCFC tested consecutive doses of 3, 10, 30, 100, 300, and 600 mg of peanut protein, given 20 to 30 minutes apart, as tolerated with no or mild symptoms.
In the intention-to-treat (ITT) analysis, 23 of 29 AR101 subjects (79 percent) tolerated a single highest dose of at least 300 mg of peanut protein, versus 5 of 26 placebo subjects (19 percent) (p<0.0001). Additionally, 18 of 29 AR101 subjects (62 percent) tolerated a single highest dose of 600 mg of peanut protein, versus 0 of 26 placebo subjects (0 percent) (p<0.0001).
"The ARC001 study demonstrated that approximately six months of AR101
treatment significantly raised the level of tolerance of peanut protein,
compared to placebo," said Aimmune CEO
Of the AR101 subjects who completed the desensitization protocol (n=23), all tolerated the 300-mg dose of peanut protein. Further, 78 percent of those subjects tolerated the 600-mg dose of peanut protein, corresponding to cumulative equivalents of approximately one and a half peanuts (443 mg total) and approximately three to four peanuts (1,043 mg total), respectively.
Consistent with the known mechanisms of OIT, 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 (GI) symptoms were the most common treatment-related adverse events in both the AR101 and the placebo subjects. Recurrent GI symptoms led to the early withdrawal from treatment of four AR101 subjects and contributed, along with other factors, to the early withdrawal of two additional AR101 subjects. In all six AR101 subjects who withdrew (21 percent), the GI symptoms resolved within three weeks of discontinuing treatment.
"In our studies, we have worked to not only test AR101 as a potential
peanut allergy treatment, but also to gain understanding of peanut
allergy that could help advance the field," said
"We're hopeful that our ongoing studies of AR101 will confirm its potential as a treatment that improves upon the current standard of care, which is limited to diagnosis, avoidance of the allergenic food, and treatment of allergic reactions from accidental ingestion with epinephrine or other appropriate medication," said Dr. Adelman. "We're also grateful for the collaboration of leading food allergy oral immunotherapy investigators, and the commitment of peanut-allergic clinical trial participants and their families, as we work to establish a validated approach to treatment that could change practice and provide a reassuring level of protection to peanut-allergic patients."
1. JA Bird et al. Efficacy and safety of AR101 in oral
immunotherapy for peanut allergy: results of ARC001, a randomized,
double-blind, placebo-controlled Phase 2 clinical trial.
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 for its Phase 3 PALISADE trial
of AR101, including the timing of topline data for the trial, which is
expected in the first quarter of 2018; 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; the company's ability to initiate and/or complete clinical
trials; the unpredictability of the regulatory process; the possibility
that Aimmune's clinical trials will not be successful; Aimmune's
dependence on the success of AR101; the company's reliance on third
parties for the manufacture of the company's product candidates;
possible regulatory developments in
This press release concerns a product that is under clinical
investigation and that has not yet been approved for marketing by the