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Aimmune Therapeutics Presents Data on Biomarkers and Potential to Predict Response to Peanut Allergy Treatment With AR101

— Aimmune Discussed Phase 2 Data, Including Peanut-Specific IgE Values, at FARE Research Retreat —

BRISBANE, Calif.--(BUSINESS WIRE)--Apr. 11, 2016-- Aimmune Therapeutics, Inc. (Nasdaq:AIMT), a biopharmaceutical company developing CODIT™ (Characterized Oral Desensitization ImmunoTherapy) treatments for life-threatening food allergies, today announced that the company’s CEO, Stephen Dilly, M.B.B.S., Ph.D., presented data over the weekend as part of its participation in the annual Food Allergy Research and Education (FARE) Research Retreat. In addition to reviewing efficacy and safety data from Phase 2 trials of Aimmune’s lead product, AR101 for the treatment of peanut allergy, Dr. Dilly also discussed biomarker data from the studies.

“It was particularly pleasing to share these important findings with many of the same food allergy leaders whose vision to pursue FDA approval for an oral immunotherapy product led to the formation of our company,” said Dr. Dilly. “Potentially the most exciting new finding we presented this weekend was the very clear partitioning of patient outcomes in our Phase 2 studies based on pre-treatment baseline peanut-specific IgE (psIgE) values.”

All 55 patients who entered the ARC001 and ARC002 Phase 2 trials had documented psIgE values at pre-treatment baseline: 28 had values above 100 kUA/l and 27 had values of 100 kUA/l or less. In the 27 patients with psIgE ≤100 kUA/l, there were no treatment-related withdrawals, no serious adverse events, and no epinephrine use, and all patients met the primary endpoint at the double-blind, placebo-controlled food challenge administered at the end of up-dosing. There was a single withdrawal from this group due to scheduling issues. Conversely, in the 28 patients from the higher psIgE group, there were 10 treatment-related withdrawals and one patient failed the exit challenge, giving an overall treatment success rate of 61 percent in this group.

Other baseline measures, including skin prick test and maximum tolerated dose at entry double-blind, placebo-controlled food challenge, were not conspicuously different between patients who completed up-dosing and those who discontinued treatment during up-dosing.

“If replicated in our ongoing Phase 3 PALISADE trial, we believe that these findings could have very positive implications for the adoption of AR101 in clinical practice,” continued Dr. Dilly. “According to the published scientific literature, at least 80 percent of untreated people with peanut allergy have psIgE levels below 100 kUA/l, and, importantly, there seems to be no clear correlation between psIgE levels and reaction threshold or severity of reaction.”

“Our Phase 2 data suggest that peanut-specific IgE levels could play an important role in identifying patients at increased risk for side effects, particularly gastrointestinal side effects, from oral immunotherapy. We are eager to see if this pattern around peanut-specific IgE levels will be confirmed in our Phase 3 PALISADE trial. If it is, we believe that a simple predictive test could ultimately help guide the course of oral immunotherapy,” said Aimmune’s Chief Medical Officer, Robert Elfont, M.D., Ph.D. “Although patients with high peanut-specific IgE levels, in Phase 2, had a rockier course with up-dosing, it is interesting to note that the efficacy signal in completers was similar across all patients, independent of psIgE levels.”

As previously reported, treatment with AR101 was associated with significantly increased peanut-specific IgG4 antibodies and a significantly decreased ratio of psIgE to IgG4. Also, the median peanut skin prick test wheal size declined meaningfully from measurements taken before treatment to those taken after patients had completed the AR101 up-dosing regimen.

In a separate presentation at the Research Retreat, Erik Wambre, Ph.D., of the Benaroya Research Institute (BRI) discussed research from his lab focusing on peanut-specific Th2 lymphocytes. His talk included data from a small pilot study of participants in Aimmune’s Phase 2 trials, which he previously discussed at the 2016 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in March. In these preliminary studies, activated peanut-specific Th2 cells were greatly reduced in the blood of subjects receiving active treatment, but not placebo, suggesting that AR101 specifically modulated key lymphocyte populations responsible for peanut allergy. Aimmune and BRI have an ongoing partnership and plan to expand these and other related studies during the Phase 3 PALISADE trial, with generous support from the Immune Tolerance Network.

“These data are extremely encouraging because they demonstrate that the reduction in clinical reactivity following treatment with AR101 is clearly linked to reduction of the pathogenic T cells associated with peanut allergy, and other quantifiable changes in the immune repertoire,” said Dr. Wambre. “We look forward to better understanding the ability of AR101 to reprogram the immune system by expanding our studies — not only in T cells but also B cells, basophils, and antibodies — in many more participants from Aimmune’s ongoing Phase 3 trial. It is exciting to be a part of the biggest peanut immunotherapy study ever undertaken because the large sample will give us unprecedented power to detect biomarkers of treatment response.”

About Food Allergies

Food allergies are a significant and growing health problem in the United States, Europe and throughout the developed world. It is estimated that more than 30 million people in the United States and Europe have a food allergy, and more than five million people in the United States and Europe have peanut allergy, including more than two million children. The prevalence of peanut allergy in children in the United States is estimated to have tripled between 1997 and 2008, and experts believe it has continued to rise since 2008. For people living with food allergies, certain foods can cause severe allergic reactions, including potentially life-threatening anaphylaxis. There are no approved medical therapies to cure food allergies or prevent their effects. Currently, food-allergic patients manage their condition by strict allergen avoidance and carrying epinephrine auto-injectors for use in case of accidental exposure. Thus, in addition to the unmet medical need, food allergies can impose a significant quality of life burden. For more information, please see www.foodallergy.org and www.niaid.nih.gov/topics/foodallergy.

About AR101 and CODIT™

Aimmune Therapeutics is developing AR101 as a potential desensitization therapy for patients with peanut allergy to provide them with protection from reactions to peanut allergens at a level believed to substantially exceed the amount typically encountered in an accidental exposure. AR101 maintains the complete range of natural peanut proteins, which are rigorously analyzed and combined with pharmaceutical-grade ingredients to ensure that each dose has consistent amounts of peanut protein with well-defined concentrations of peanut allergens, especially the three key allergenic proteins (Ara h1, h2 and h6). Patients ingest AR101 mixed into small amounts of palatable, age-appropriate food.

AR101 is part of Aimmune’s approach to treating food allergies using its Characterized Oral Desensitization ImmunoTherapy, or CODIT™, system. The CODIT system leverages extensive independent scientific research on oral immunotherapy, or OIT, demonstrating that food allergy patients can be desensitized to food allergens by being administered well-defined, gradually increasing doses of the allergen over a period of months. Aimmune’s CODIT system is designed to precisely control the amounts of the allergens administered in a systematic dosing regimen, beginning with very low doses of the allergens. Once a patient attains a clinically meaningful level of desensitization, the patient continues to take a daily maintenance dose of the CODIT system product in order to maintain the desensitization.

About Aimmune’s Phase 2 and Phase 3 Clinical Trials

Of the 55 patients who entered Aimmune’s Phase 2 trials (ARC001 and the ARC002 rollover/crossover), 44 patients completed the approximately six-month up-dosing period to a daily dose of 300 mg of AR101. At the end of that period, 43 of those patients tolerated a cumulative amount of 443 mg of peanut protein in a double-blind, placebo-controlled food challenge (DBPCFC). Additionally, 35 of the patients who completed up-dosing tolerated a cumulative amount of 1,043 mg of peanut protein in the DBPCFC, the highest challenge administered at that point. The patients who tolerated at least 443 mg of peanut protein in the post–up-dosing DBPCFC were eligible to continue on 300 mg of AR101 per day in maintenance therapy. After three months of maintenance, the patients (n=40) underwent another DBPCFC, where 100 percent, 90 percent, and 60 percent of patients tolerated cumulative amounts of peanut protein of 443 mg, 1,043 mg, and 2,043 mg, respectively (corresponding to 72 percent, 65 percent, and 44 percent on an intent-to-treat basis with n=55).

Approximately 90 percent of the treatment-related adverse events in Phase 2 have been mild, predominantly allergic, symptoms, consistent with stimulation of the immune system. There have been no treatment-related severe adverse events. One serious adverse event of moderate, non-life-threatening anaphylaxis occurred in Phase 2 early in the course of up-dosing. Ten patients discontinued from the trial for treatment-related reasons, all due directly or indirectly to gastrointestinal adverse events experienced early in the up-dosing regimen. These events typically occurred within the first few weeks of the treatment, and in all cases the symptoms resolved within three weeks of the cessation of treatment.

The primary endpoint in Aimmune’s currently enrolling Phase 3 PALISADE trial of AR101 is tolerating a cumulative amount of at least 1,043 mg of peanut protein after approximately six months of up-dosing and six months of maintenance therapy at a daily dose of 300 mg of AR101. PALISADE is a randomized 3:1, double-blind, placebo-controlled trial expected to enroll approximately 500 peanut-allergic patients 4-55 years of age at more than 60 clinical sites in the United States, Canada, and nine countries in the European Union.

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™) system, an approach to oral immunotherapy (OIT), uses rigorously characterized product candidates with gradual, controlled up-dosing protocols to obtain clinically meaningful desensitization to food allergens. Aimmune’s first CODIT product, 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. Aimmune’s Phase 3 trial of AR101, PALISADE, is now enrolling patients. 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 development efforts; Aimmune’s expectations regarding the potential benefits of AR101; Aimmune’s ability to use biomarkers to predict prospectively how patients will react to AR101; Aimmune’s expectations regarding expanding its partnership with BRI and conducting additional studies; and Aimmune’s expectations regarding potential applications of its CODIT™ system. Risks and uncertainties that contribute to the uncertain nature of the forward-looking statements include: Aimmune’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 reliance on third parties for the manufacture of its product candidates and the conduct of its Phase 3 clinical trial for 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 the Annual Report on Form 10-K for the period ending December 31, 2015, filed on March 3, 2016. 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.

Source: Aimmune Therapeutics, Inc.

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Aimmune Therapeutics, Inc.
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