Aimmune’s PALFORZIA®▼ [defatted powder of Arachis hypogaea L., semen (peanuts)] Receives Final Appraisal Determination (FAD) as Positive Draft Guidance From NICE for the Treatment of Peanut Allergy in Patients Aged 4 to 17 Years

– First peanut allergy treatment licensed and available to patients living in England

– National Institute for Health and Care Excellence (NICE) decision is based on results from the PALISADE, ARTEMIS and the ARC004/PALISADE follow-on clinical studies

– Peanut allergy affects between 0.5-2.5% of children in the UK¹

LONDON–(BUSINESS WIRE)–Aimmune Therapeutics UK Ltd., a biopharmaceutical company developing and commercialising pharmaceutical therapies to prevent, manage, and treat food and metabolic related diseases, today announced that PALFORZIA^® [defatted powder of Arachis hypogaea L., semen (peanuts)] has received the Final Appraisal Determination (FAD) as positive draft guidance for use within the National Health Service (NHS) for the treatment of peanut allergy in patients aged 4 to 17. PALFORZIA is indicated in patients aged 4 to 17 years with a confirmed diagnosis of peanut allergy in conjunction with a peanut-avoidant diet and may be continued in patients 18 years of age and older.

In the UK, peanut allergy affects between 0.5-2.5% of children.² Unlike other common food allergies, peanut allergy can be lifelong.³ Reactions to peanut are potentially life-threatening, accounting for the majority of deaths related to food allergy.⁴ The standard of care has been a strict elimination diet and the timely administration of rescue medications in case of an allergic reaction from accidental exposure.⁵ Despite vigilance, accidental exposures may occur and cause reactions of unpredictable severity, leading to a lifelong risk of severe reactions.⁶ Beyond the physical impact of peanut allergy, this condition also has a significant psychosocial impact on children, their families and caregivers. Those living with peanut allergy face many uncertainties and restrictions, in addition to constant feelings of fear, frustration, anxiety and stress in their daily lives.⁷

“This is excellent news. We know all too well that peanut allergy is extremely challenging and stressful for most children and teenagers with this condition. New treatment options are essential to mitigate the risk of potential severe allergic reactions due to accidental exposure,” said Simon Williams, Chief Executive Officer, Anaphylaxis Campaign, a UK-wide charity for people at risk of severe allergies. “The availability of PALFORZIA through NHS England is a significant step forward for people living with peanut allergy.”

“Results from the phase 3 clinical trials and additional studies demonstrate the potential of PALFORZIA to mitigate against severe allergic reactions in the event of accidental exposure to peanut protein,” said Dr Mich Erlewyn-Lajeunesse, a Consultant in Paediatric Allergy at Southampton Children’s Hospital and study investigator for the PALISADE and ARTEMIS trials. “As the first peanut allergy treatment available to patients on NHS England, today’s announcement is a very important milestone in our journey to bringing PALFORZIA to as many eligible patients across England as possible.”

The positive draft recommendation is based on positive data from two phase 3 studies – the pivotal PALISADE (NCT02635776) study conducted in North America and Europe and the ARTEMIS (NCT03201003) study that focused on Europe.^{8 9 10} Both studies met primary, secondary and safety endpoints for patients aged 4 to 17 and demonstrated that patients treated with PALFORZIA experienced desensitization to peanut with an anticipated and manageable safety profile.¹¹ The recommendation is also based on the ARC004 (NCT02993107) PALISADE open-label follow-on study, which demonstrated the importance of long-term daily maintenance (up to 24 months) dosing with PALFORZIA.¹² Additionally, results from a cross-sectional, observational study using different data collection methods, conducted in the UK, provided the estimates of quality of life (QoL) utility measures for patients and caregivers across different health states relating to oral immunotherapy (OIT). The data showed a gain in utility related to QoL among patients (aged 4 to 17 years) treated with OIT for peanut allergy and their caregivers between untreated peanut allergy and post-treated peanut allergy OIT health states.¹³

“We are very proud to have worked with NICE and the peanut allergy community to make PALFORZIA available in early 2022 to patients aged 4 to 17 with a confirmed diagnosis of peanut allergy,” said Nicolas Fouché, Senior Vice-President & Head, International, Aimmune Therapeutics. “The swift and positive draft recommendation by NICE’s Appraisal Committee reinforces our belief that PALFORZIA is a truly innovative and effective medicine to treat patients aged 4 to 17 living with peanut allergy. This long-awaited treatment option has an important role to play in allowing these patients to live with far fewer restrictions and reduce the risk of severe allergic reactions due to accidental peanut exposure.”

About the PALISADE and ARTEMIS phase 3 clinical trials

PALISADE (Peanut Allergy Oral Immunotherapy Study of AR101 for Desensitization) and ARTEMIS (AR101 Trial in Europe Measuring Oral Immunotherapy Success) evaluated the efficacy and safety of PALFORZIA in 671 participants aged 4 to 17 with peanut allergy in North America and Europe.

Participants enrolled represented a highly allergic population with a high prevalence of allergy-related comorbidities who reacted to low doses of peanut protein at the screening double-blind, placebo-controlled food challenge (DBPCFC). Participants underwent an up-dosing period for 20 to 40 weeks starting at 3 mg until the 300 mg dose was reached. Participants then underwent six months (PALISADE) or three months (ARTEMIS) of maintenance immunotherapy with 300 mg PALFORZIA or placebo until the end of the study.

The primary efficacy endpoint in both studies was the proportion of participants who tolerated a single highest dose of at least 1,000 mg peanut protein (equivalent to seven peanut kernels cumulatively) with no more than mild allergic symptoms at the exit challenge. Key secondary endpoints included desensitization response rates after single doses of 300 mg and 600 mg peanut protein and the maximum severity of symptoms at the exit challenge.

About PALISADE-ARC004

PALISADE-ARC004 aimed to explore the impact of continued PALFORZIA therapeutic maintenance dosing (300 mg/day) on efficacy, safety/tolerability, and food allergy-related quality of life. This analysis focuses on a subset of PALISADE participants who completed treatment with PALFORZIA, tolerated ≥300 mg peanut protein (443 mg cumulative) at PALISADE exit double-blind, placebo-controlled food challenge (DBPCFC), and enrolled in ARC004. Participants received daily therapeutic maintenance dosing 300 mg/day for 28 weeks (Group A) or 56 weeks (Group B) accounting for a total of ~1.5 and 2 years of treatment respectively including PALISADE. This analysis included 142 participants ages 4 to 17 years. Two participants in these cohorts turned 18 during PALISADE and were included in this analysis but not in the ARC004 analyses.

About ARC004

ARC004 was an open-label follow-on study to the phase 3 PALISADE trial and assessed the safety and efficacy of extended daily and non-daily dosing of PALFORZIA across 351 peanut-allergic patients ages 4 to 17 years. Participants who received placebo (PALFORZIA-naïve) in PALISADE received active treatment with PALFORZIA, and eligible participants who received PALFORZIA in PALISADE (PALFORZIA continued) were assigned to either daily or non-daily continued treatment regimen with different durations. A total of 261 PALFORZIA-treated participants completed the ARC004 study.

About PALFORZIA

PALFORZIA was approved in December 2020 by the European Commission (EC) for patients aged 4 to 17 years with a confirmed diagnosis of peanut allergy in conjunction with a peanut-avoidant diet. PALFORZIA may be continued in patients 18 years of age and older.

The conversion of the EU Centralised Procedure to Great Britain Marketing Authorisation was validated by the MHRA (Medicines and Healthcare products Regulatory Agency) in April 2021.

PALFORZIA is a complex biologic drug used with a structured dosing approach that builds on a century of oral immunotherapy (OIT) research. With OIT, the specific allergenic proteins are ingested initially in very small quantities, followed by incrementally increasing amounts, that can result in the ability to mitigate allergic reactions to the allergen over time. PALFORZIA is a rigorously developed, pharmaceutical-grade OIT for peanut allergy with a well-defined allergen profile to assure the consistency of every dose, from 0.5 mg (equivalent to approximately 1/600^th of a peanut kernel) to 300 mg.

PALFORZIA is not intended for, and does not provide, immediate relief of allergic symptoms. Therefore, this medicinal product is not to be used for emergency treatment of allergic reactions, including anaphylaxis. Self-injectable adrenaline (epinephrine) must be available to the patient at all times.

For more information, please see the PALFORZIA full Summary of Product Characteristics (SmPC) at www.aimmune.co.uk/our-product.

About Aimmune Therapeutics UK Ltd.

Aimmune Therapeutics UK Ltd. is a biopharmaceutical company developing and commercialising pharmaceutical therapies to prevent, manage, and treat food and metabolic related diseases. For more information, please visit www.aimmune.co.uk.

▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. If you experience any side effects, talk to your doctor, pharmacist or nurse. This includes any side effects not listed in the package leaflet. You can also report side effects directly via the Yellow Card Scheme at https://yellowcard.mhra.gov.uk/. By reporting side effects, you can help provide more information on the safety of this medicine.

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¹ Stiefel, G., Anagnostou, K., Boyle, R.J., et al. 2017. BSACI Guideline for the Diagnosis and Management of Peanut and Tree Nut Allergy. Clinical and Experimental Allergy. 47, pp. 719-739.

² Stiefel, G., Anagnostou, K., Boyle, R.J., et al. 2017. BSACI Guideline for the Diagnosis and Management of Peanut and Tree Nut Allergy. Clinical and Experimental Allergy. 47, pp. 719-739.

³ Du Toit G, Katz Y, Sasieni P, Mesher D, Maleki SJ, Fisher HR, Fox AT, Turcanu V, Amir T, Zadik-Mnuhin G, Cohen A, Livne I, Lack G. Early consumption of peanuts in infancy is associated with a low prevalence of peanut allergy. J Allergy Clin Immunol. 2008 Nov;122(5):984-91. doi: 10.1016/j.jaci.2008.08.039. PMID: 19000582.

⁴ Bock SA, Muñoz-Furlong A, Sampson HA. Fatalities due to anaphylactic reaction to foods. J Allergy Clin Immunol. 2001;107:191.

⁵ Lanser, Bruce J., et al. “Current options for the treatment of food allergy.” Pediatric Clinics 62.6 (2015): 1531-1549.

⁶ Lanser, Bruce J., et al. “Current options for the treatment of food allergy.” Pediatric Clinics 62.6 (2015): 1531-1549.

⁷ DunnGalvin, A., et al. APPEAL-1: A multiple-country European survey assessing the psychosocial impact of peanut allergy. Allergy. 2020 Nov;75(11):2899-2908. DOI: 10.1111/all.14363. Epub 2020 Jul 9.PMID: 32400915

⁸ PALFORZIA. Summary of Product Characteristics.

⁹ Vickery BP, et al. AR101 oral immunotherapy for peanut allergy. New Engl J Med 2018; DOI: 10.1056/NEJMoa1812856

¹⁰ Hourihane JO, et al. Efficacy and safety of oral immunotherapy with AR101 in European children with a peanut allergy (ARTEMIS): a multicentre, double-blind, randomised, placebo-controlled phase 3 trial. Lancet Child & Adolescent Health. 2020; 4:10: 728-739.

¹¹ Vickery BP, et al. AR101 oral immunotherapy for peanut allergy. New Engl J Med 2018; DOI: 10.1056/NEJMoa1812856

¹² Fernandez-Rivas, M, et al. Open-label follow-on study evaluating the efficacy, safety, and quality of life with extended daily oral immunotherapy in children with peanut allergy. Allergy: European Journal of Allergy and Clinical Immunology 2021; DOI: 10.1111/all.15027

¹³ Poster #P20: Gallop, K, et al. “Exploring health-related quality of life burden in peanut allergy and the potential benefit of oral immunotherapy.” PAAM 2021

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