Aimmune Therapeutics to Present Positive Results from Phase 3 POSEIDON Study of PALFORZIA® [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in Peanut-Allergic Children Aged 1 to 3 Years

— Results Will be Presented for the First Time at the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting —

— 73.5% of Patients Treated with PALFORZIA Met the Primary Outcome at 12 Months Compared with 6.3% of Patients in the Placebo Arm —

— 61% of Patients Treated with PALFORZIA Tolerated 2,000 mg of Peanut Protein During the 12-Month Exit Food Challenge —

— 2% of Patients Treated with PALFORZIA Experienced PALFORZIA-Related Systemic Allergic Reactions —

BRISBANE, Calif.--(BUSINESS WIRE)--Aimmune Therapeutics, Inc., a Nestlé Health Science company developing pharmaceutical therapies to prevent, manage, and treat food, gastrointestinal (GI), and metabolic-related diseases, will present for the first time results from the Phase 3 POSEIDON (Peanut Oral Immunotherapy Study of Early Intervention for Desensitization) study. The trial evaluated the efficacy and safety of PALFORZIA^® [Peanut (Arachis hypogaea) Allergen Powder-dnfp] in peanut-allergic children aged 1 to 3 years. These findings will be presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific Meeting in Louisville, Kentucky on Saturday, November 12, 2022.

The study met the primary outcome, with the majority of patients treated with PALFORZIA tolerating at least a 600 mg single dose (1,043 mg cumulative) or 1,000 mg single dose (2,043 mg cumulative) of peanut protein (73.5% and 68.4%, respectively, compared to 6.3% and 4.2% of patients who received placebo, respectively). In addition, the majority of patients treated with PALFORZIA (61.2% compared with 2.1% of placebo-treated patients) tolerated the highest exit double-blind, placebo-controlled exit food challenge (DBPCFC) dose level of 2,000 mg (4,043 mg cumulative), and the median highest tolerated dose of peanut increased 66-fold between baseline and exit DBPCFC.

PALFORZIA demonstrated a favorable safety profile. Overall, 84.7% of patients on PALFORZIA and 93.8% of patients on placebo completed the study. There were no PALFORZIA-related serious or severe adverse events (AEs), and systemic allergic reactions due to any cause were similar between PALFORZIA and placebo-treated patients (8.1% and 8.3%, respectively). Treatment-related systemic allergic reactions occurred in 2% of PALFORZIA-treated patients and 0% of placebo-treated patients.

“These results indicate that treatment with PALFORZIA in young children resulted in a clinically meaningful change in the amount of peanut they could tolerate during oral challenge,” said Stephen Tilles, M.D., Senior Medical Director and Global Head of Medical Affairs, Allergy for Aimmune Therapeutics. “Currently, there is a high unmet need for these young children with peanut allergy who rely on avoidance alone, and treatment at this early age presents a unique window of opportunity to actively manage their peanut allergy near the time of diagnosis.”

Peanut allergy is one of the most common food allergies in the world, affecting more than 1.6 million children and teens in the U.S. ⁱ Reactions to peanut are potentially life-threatening, accounting for the majority of deaths related to food allergy.ⁱⁱ

“In my treatment of children aged 4 through 17 with PALFORZIA, I have seen firsthand the benefits of this therapy,” said George du Toit, M.B., B.Ch., Professor of Pediatric Allergy at Evelina London Children’s Hospital, Guy's and St Thomas' NHS Foundation Trust, King’s College London and study investigator in PALFORZIA clinical trials. “I am now encouraged by these promising results in younger children, which show that the majority of study participants tolerated a dose of 600mg of peanut protein – the equivalent of two peanut kernels – after 12 months of treatment, and no severe systemic allergic reactions and only infrequent mild-moderate systemic allergic reactions reported among PALFORZIA-treated participants.”

“Parents of children with peanut allergy may not be able to constantly supervise them outside of the home to avoid accidental exposure to peanut,” said Amy M. Scurlock, M.D., Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children’s Hospital. “The encouraging results of the POSEIDON study indicate that PALFORZIA has the potential to be an effective and convenient treatment option for peanut-allergic children aged 1 to 3 years. These outcomes are welcome news for the many parents and young children impacted by this condition.”

PALFORZIA is currently approved by the FDA as an oral immunotherapy (OIT) for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut in patients aged 4 through 17 years with a confirmed diagnosis of peanut allergy.

Aimmune plans to submit the full POSEIDON study results for publication in a peer-reviewed journal and submit these study results to the FDA in the first half of 2023 to support the use of PALFORZIA in appropriate peanut-allergic children aged 1-3 years, subject to review and approval by FDA.

POSEIDON Results

POSEIDON enrolled 146 patients, with 98 randomized to PALFORZIA and 48 randomized to placebo. Overall, 84.7% of patients on PALFORZIA and 93.8% of patients on placebo completed the study.

Efficacy results:

• 73.5% and 68.4% of patients treated with PALFORZIA tolerated a single dose of 600 mg and 1,000 mg of peanut protein compared with 6.3% and 4.2% of patients in the placebo group, respectively, with a treatment difference of 67.2% and 64.2% (p<0.0001 for both).
• 61.2% of PALFORZIA-treated patients tolerated the highest dose level in the exit DBPCFC of 2,000 mg (cumulative 4,043 mg) compared with 2.1% of placebo-treated patients, with a treatment difference of 59.1% (p<0.0001).

Key safety results:

• The most common AEs related to PALFORZIA treatment involved the skin and subcutaneous tissues (urticaria, erythema), the gastrointestinal system (abdominal pain, vomiting), and the respiratory, thoracic and mediastinal system (cough, sneezing).
• 5.1% of PALFORZIA-treated patients versus 2.1% of placebo-treated patients experienced early withdrawal due to AEs.
• 6.1% of PALFORZIA-treated patients versus 4.2% of placebo-treated patients experienced serious AEs. None were related to treatment.
• 2% of PALFORZIA-treated patients versus 0% of placebo-treated patients experienced systemic allergic reactions (SARs; anaphylactic reactions of any severity) related to treatment. SARs are defined according to the National Institute of Allergy and Infectious Disease criteria and graded using the 3-point Muraro (European Academy of Allergy and Clinical Immunology) grading scale.
• SARs were similar between PALFORZIA-treated and placebo-treated patients (8.1% and 8.3%, respectively).
• There were no severe or serious systemic allergic reactions.
• 3.1% of PALFORZIA-treated patients versus 0% of placebo-treated patients used epinephrine for a treatment-related AEs.

Abstracts to be presented by Aimmune at the ACAAI conference include:

• Oral Immunotherapy In Children Aged 1 To <4 Years With Peanut Allergy: POSEIDON Trial Outcomes, Saturday, November 12, 4:43-4:53 p.m. ET
• Health-Related Quality of Life in Children and Caregiver Proxies from Trials of Peanut Oral Immunotherapy, Saturday, November 12, 5:13-5:23 p.m. ET
• Manufacturing Processes and Standardization of Allergen Potency of Peanut (Arachis hypogaea) Allergen Powder-dnfp, Friday, November 11, 4:00-4:15 p.m. ET
• Oral Immunotherapy in Children with Peanut Allergy Alone or with Other Food Allergy in PALISADE, Friday, November 11, 4:15-4:30 p.m. ET
• Patient and caregiver perspectives on treatment with licensed peanut allergy oral immunotherapy in practice, Friday, November 11, 3:15-3:30 p.m. ET

About POSEIDON

POSEIDON (Peanut Oral Immunotherapy Study of Early Intervention for Desensitization, clinicaltrials.gov number NCT03736447) is an international, randomized (2:1), double-blind, placebo-controlled Phase 3 trial that evaluated the efficacy and safety of PALFORZIA in peanut-allergic children aged 1 to 3 in North America and four European countries. Enrollment was based on stringent entry criteria, including a documented clinical history of peanut allergy, positive skin prick tests and/or elevated blood levels of peanut antibodies, and dose-limiting symptoms after consuming single doses of peanut protein >3 to ≤300 mg in a positive double-blind, placebo-controlled food challenge (DBPCFC). In POSEIDON, patients underwent a dose-escalation period of approximately 22 weeks to reach a dose of 300 mg per day of PALFORZIA or placebo, then continued on that dose for approximately six months. At the end of the trial, patients underwent an exit DBPCFC.

About PALFORZIA^® [Peanut (Arachis hypogaea) Allergen Powder-dnfp]

PALFORZIA was approved in January 2020 by the U.S. Food and Drug Administration (FDA) as an oral immunotherapy (OIT) for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut in patients aged 4 through 17 years with a confirmed diagnosis of peanut allergy. PALFORZIA was approved on December 17, 2020 by the European Commission (EC), on April 7, 2021 by the Medicines and Healthcare products Regulatory Agency (MHRA) in the U.K., and on May 4, 2021 by Swissmedic. Use of PALFORZIA may be continued in patients 18 years of age and older. PALFORZIA is not indicated for the emergency treatment of allergic reactions, including anaphylaxis and must always be used in conjunction with a peanut-avoidant diet.

Boxed WARNING: PALFORZIA can cause anaphylaxis, which may be life threatening and can occur at any time during PALFORZIA therapy. Prescribe injectable epinephrine, instruct and train patients on its appropriate use, and instruct patients to seek immediate medical care upon its use. Do not administer PALFORZIA to patients with uncontrolled asthma.

Please see U.S. Important Safety Information and Boxed WARNING below, or for full Prescribing Information, including Boxed WARNING and Medication Guide, visit www.PALFORZIA.com.

INDICATION

PALFORZIA is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut. PALFORZIA is approved for use in patients with a confirmed diagnosis of peanut allergy. Initial Dose Escalation may be administered to patients aged 4 through 17 years. Up-Dosing and Maintenance may be continued in patients 4 years of age and older.

PALFORZIA is to be used in conjunction with a peanut-avoidant diet.

Limitations of Use: Not indicated for the emergency treatment of allergic reactions, including anaphylaxis.

IMPORTANT SAFETY INFORMATION

Boxed WARNING:

PALFORZIA can cause anaphylaxis, which may be life threatening and can occur at any time during PALFORZIA therapy.

Prescribe injectable epinephrine, instruct and train patients on its appropriate use, and instruct patients to seek immediate medical care upon its use.

Do not administer PALFORZIA to patients with uncontrolled asthma.

Dose modifications may be necessary following an anaphylactic reaction.

Observe patients during and after administration of the Initial Dose Escalation and the first dose of each Up-Dosing level, for at least 60 minutes.

PALFORZIA is available only through a restricted program called the PALFORZIA REMS.

CONTRAINDICATIONS

PALFORZIA is contraindicated in patients with uncontrolled asthma, or with a history of eosinophilic esophagitis and other eosinophilic gastrointestinal disease.

WARNINGS AND PRECAUTIONS

Anaphylaxis

PALFORZIA can cause anaphylaxis, which may be life threatening. PALFORZIA is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the PALFORZIA REMS because of the risk of anaphylaxis. Only prescribers, healthcare settings, pharmacies, and patients certified and enrolled in the REMS Program can prescribe, receive, dispense or administer PALFORZIA.

Anaphylaxis has been reported during all phases of PALFORZIA dosing, including Maintenance and in subjects who have undergone recommended Up-Dosing and dose modification procedures.

Do not initiate PALFORZIA treatment in a patient who has had severe or life-threatening anaphylaxis within the previous 60 days. PALFORZIA may not be suitable for patients with certain medical conditions that may reduce the ability to survive anaphylaxis, including but not limited to markedly compromised lung function, severe mast cell disorder, or cardiovascular disease. In addition, PALFORZIA may not be suitable for patients taking medications that can inhibit or potentiate the effects of epinephrine.

All Initial Dose Escalation doses and the first dose of each Up-Dosing level must be administered in a certified health care setting.

Patients may be more likely to experience allergic reactions following PALFORZIA administration in the presence of cofactors such as exercise, hot water exposure, intercurrent illness (e.g., viral infection), or fasting. Other potential cofactors may include menstruation, sleep deprivation, nonsteroidal anti-inflammatory drug use, or uncontrolled asthma. Patients should be proactively counseled about the potential for the increased risk of anaphylaxis in the presence of these cofactors. If possible, adjust the time of dosing to avoid these cofactors. If it is not possible to avoid these cofactors, consider withholding PALFORZIA temporarily.

Asthma

Uncontrolled asthma is a risk factor for a serious outcome, including death, in anaphylaxis. Ensure patients with asthma have their asthma under control prior to initiation of PALFORZIA.

PALFORZIA should be temporarily withheld if the patient is experiencing an acute asthma exacerbation. Following resolution of the exacerbation, resumption of PALFORZIA should be undertaken cautiously. Re-evaluate patients who have recurrent asthma exacerbations and consider discontinuation of PALFORZIA.

Eosinophilic Gastrointestinal Disease

Discontinue PALFORZIA and consider a diagnosis of eosinophilic esophagitis in patients who experience severe or persistent gastrointestinal symptoms, including dysphagia, vomiting, nausea, gastroesophageal reflux, chest pain, or abdominal pain.

Gastrointestinal Adverse Reactions

Gastrointestinal adverse reactions were commonly reported in PALFORZIA-treated subjects, and dose modification should be considered for patients who report these reactions. For severe or persistent gastrointestinal symptoms consider a diagnosis of eosinophilic esophagitis.

ADVERSE REACTIONS

The most common adverse events reported in subjects treated with PALFORZIA (incidence ≥ 5% and ≥ 5% than placebo) are abdominal pain, vomiting, nausea, oral pruritus, oral paresthesia, throat irritation, cough, rhinorrhea, sneezing, throat tightness, wheezing, dyspnea, pruritus, urticaria, anaphylactic reaction, and ear pruritus.

For more information about PALFORZIA, please call 1-844-PALFORZ (1-844-725-3679) or visit www.PALFORZIA.com.

Please see full U.S. Prescribing Information, including Boxed WARNING, and Medication Guide at www.PALFORZIA.com.

About Aimmune Therapeutics

Aimmune Therapeutics, part of Nestlé Health Science, is a biopharmaceutical company that aspires to be a global leader in developing innovative medicines to prevent, manage, and treat food, gastrointestinal (GI), and metabolic-related diseases for those with few or no treatment options. Aimmune was built on the belief that families deserve standardized, regulatory-approved, science-driven treatments that reduce their daily burden and help them better manage these conditions.

For more information about Aimmune, please visit www.aimmune.com

About Nestlé Health Science

Nestlé Health Science, a leader in the science of nutrition, is a globally managed business unit of Nestlé. We are committed to redefining the management of health, offering an extensive portfolio of science-based consumer health, medical nutrition, pharmaceutical therapies, and vitamin and supplement brands. Our extensive research network provides the foundation for products that empower healthier lives through nutrition. Headquartered in Switzerland, we have more than 12,000 employees around the world, with products available in more than 140 countries.www.nestlehealthscience.com

ⁱ Pawankar R, Canonica WG, Holgate ST, Lockey RF, Blaiss MS. Executive Summary. In: Pawankar R, Canonica WG, Holgate ST, Lockey RF, Blaiss MS. WAO White Book on Allergy: Update 2013. Milwaukee, WI: World Allergy Organization; 2013:1-9. https://www.worldallergy.org/UserFiles/file/ExecSummary-2013-v6-hires.pdf. Accessed August 31, 2022.

ⁱⁱ Bock SA, Muñoz-Furlong A, Sampson HA. Fatalities due to anaphylactic reactions to foods. J Allergy Clin Immunol. 2001;107:191-193.

U.S. Media:
Georgina Vincent
+1.747.758.9524
gvincent@aimmune.com