Aimmune Therapeutics, Inc., a privately held biopharmaceutical company developing desensitization treatments for food allergies, announced today that a Phase 2 study (ARC001) evaluating the company's lead investigational product, AR101 for the treatment of peanut allergy, met its primary endpoint and additional endpoint of desensitizing patients to cumulative amounts of peanut protein of 443 mg and 1,043 mg, respectively. Of the 23 active-arm patients who completed the study, 100 percent tolerated exposure to 443 mg cumulative amounts of peanut protein, and 78 percent tolerated exposure to 1,043 mg cumulative amounts of peanut protein.
Wesley Burks, M.D., chairman of the department of pediatrics at the University of North Carolina School of Medicine and physician in chief of N.C. Children's Hospital, presented the results of the study today at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2015 in a late-breaking oral abstract session titled "A novel characterized peanut allergen formulation (AR101) for oral immunotherapy (OIT) induces desensitization in peanut-allergic subjects: A Phase 2 clinical safety and efficacy study."
"These results suggest that AR101 has the potential to become the first approved oral desensitization therapy for peanut allergy, a serious and growing health problem that affects more than 5 million people in the United States and Europe, including more than two million children," said Dr. Burks. "There are currently no approved drug products available for peanut allergy, and there is a need for a safe and effective treatment to help protect people from dangerous reactions triggered by trace exposures to peanut allergens."
The ARC001 Phase 2 study, conducted at eight U.S. sites, evaluated the safety and efficacy of AR101 in peanut-allergic patients ages 4-21 who failed a double-blind, placebo-controlled food challenge (DBPCFC) of less than or equal to 100 mg of peanut protein. The randomized, double-blind, placebo-controlled study had 55 patients in the intent-to-treat population, with 29 in the active group and 26 in the placebo group. The active arm had six early discontinuations due to gastrointestinal side effects (which resolved within one to three weeks after cessation of treatment) and compliance issues.
In the study, 23 of 23 patients who completed the active treatment regimen met the primary endpoint of tolerating a cumulative amount of peanut protein of at least 443 mg, compared to 5 of 26 patients receiving placebo (p?0.0001). Additionally, 18 of 23 patients who completed the active treatment regimen tolerated a cumulative amount of peanut protein of at least 1,043 mg, compared to 0 of 26 patients receiving placebo (p?0.0001). The active treatment regimen consisted of approximately 20 weeks of gradually increasing doses and two weeks of maintenance doses. For reference, one peanut kernel contains approximately 250-300 mg of peanut protein.
"We are very pleased by the results of this study and see it as an important step toward addressing the stress and anxiety patients and their caregivers feel around the possibility of an accidental exposure to peanut," said Stephen Dilly, M.B.B.S., Ph.D., chief executive officer of Aimmune Therapeutics. "These encouraging results also reinforce the potential of our CODIT™ system for the treatment of food allergies with convenient daily doses of consistent amounts of well-defined concentrations of allergen."
The majority of the adverse events observed in the active arm of the study during the treatment period were mild. One serious adverse event (SAE) involving anaphylaxis was reported in the active arm and required a single administration of epinephrine.
In the entry DBPCFC prior to the study treatment period, four patients in the active group and four patients in the placebo group had allergic reactions that required the administration of epinephrine. In the exit DBPCFC following the study treatment period, two patients in the active group had allergic reactions requiring the administration of epinephrine, compared to 11 patients in the placebo group who had allergic reactions requiring the administration of epinephrine, including three patients who required two doses. In the active group, both uses of epinephrine at exit were triggered by moderate reactions at the highest challenge dose of 600 mg, whereas in the placebo group, the uses of the epinephrine at exit were triggered by moderate or severe reactions at doses as low as 30 mg. The exit DBPCFC dose progression was: 3 mg, 10 mg, 30 mg, 100 mg, 300 mg, 600 mg, with the 300 mg dose corresponding to the cumulative dose of 443 mg and the 600 mg dose corresponding to the cumulative dose of 1,043 mg.
Patients who completed ARC001 were eligible to participate in ARC002, an open-label study designed to evaluate the long-term safety, efficacy and tolerability of AR101.
Aimmune Therapeutics is developing AR101 as a potential desensitization therapy for patients with peanut allergy to provide them with protection from peanut allergens at a level that substantially exceeds the amount typically encountered in an accidental exposure. AR101, which has been granted "Fast-Track" designation by the U.S. Food and Drug Administration, is a complex mixture of naturally occurring proteins and pharmaceutical-grade ingredients designed to enable the convenient dosing of consistent amounts of peanut protein with well-defined concentrations of peanut allergens. Patients ingest AR101 mixed with a common age-appropriate food.
AR101 is part of Aimmune Therapeutics' 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 exposure to food allergens by being administered well-defined, gradually increasing doses of the allergen over a period of months. Aimmune Therapeutics' CODIT system is designed to precisely control the amount of allergen administered and follow a systematic dosing regimen that begins with very low doses of the allergen. Once a patient attains desensitization to a clinically meaningful level of food allergen, the patient continues to take a daily maintenance dose of the CODIT system product in order to maintain such desensitization.
Aimmune Therapeutics plans to initiate a Phase 3 registration trial of AR101 in children and adults with peanut allergies and Phase 2 studies of CODIT product candidates for two additional food allergies.