Corporate Presentation. October 2018

Transcription

1 Corporate Presentation October 2018

2 Forward-Looking Statements This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995 regarding, among other things, clinical development plans, anticipated milestones, product candidate benefits, potential market size, product adoption, market positioning, competitive strengths, product development, and other clinical, business and financial matters. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially. Risks and uncertainties include, but are not limited to, our limited operating history, our need for additional financing to achieve our goals, our dependence on our lead product AR101, the need for additional clinical testing of AR101, uncertainties relating to the regulatory process, uncertainties relating to the timing and operation of clinical trials, potential safety issues, possible lack of market acceptance of our product candidates, the intense competition in the biopharmaceutical industry, our dependence on exclusive third-party suppliers and manufacturers, and limitations on intellectual property protection. A further list and description of these risks, uncertainties and other factors can be found in our report on Form 10-Q filed on August 8, Copies of this filing are available online at or Any forward-looking statements made in this presentation speak only as of the date of the presentation. We do not undertake to update any forward-looking statements as a result of new information or future events or developments. 2

3 Aimmune Therapeutics Today Addressing a Large Unmet Need in Food Allergy CODIT Proprietary Platform Approach AR101: Potentially the First Approved Treatment for Peanut Allergy Millions of people with life-threatening food allergies No approved therapies FDA-regulated biologic medications with characterized allergen profiles and convenient, once daily oral dosing, plus education, training and support Landmark Phase 3 trial met primary and key secondary endpoints in peanut allergy, ages 4-17 Strong proprietary position including biologic data exclusivity, issued patents, and exclusive commercial supply Specialty launch to allergists can be efficiently executed Planning for Success Potential first approval and availability of AR101 in

4 There Is a Large and Growing Need for Food Allergy Treatments >30 million people in the United States and Europe have food allergies 1-4 Every 3 minutes, a food allergy reaction sends someone in the United States to the emergency room 5 ~ $4 billion in cost per year in the United States in direct medical expenses 6 No approved treatments Food Allergies Are Associated with: Constant risk and worry Inevitable accidental exposures 4 1. National Institute of Allergy and Infectious Diseases, National Institutes of Health. Retrieved from 2. United States Census Bureau Quick Facts (2015 estimates). 3. Gupta RS, et al. Pediatrics 2011;128(1):e EAACI. Food Allergy & Anaphylaxis Public Declaration. 5. Clark S et al. J Allergy Clin Immunol. 2011;127(3): Gupta RS, et al. JAMA Pediatr. 2013;167(11): Possibility of life-threatening reactions

5 Aimmune Is Developing a Comprehensive Approach to Treat Food Allergies FDA-regulated biologic medications with characterized allergen profiles Convenient, once daily oral dosing intended to gradually increase the body s ability to tolerate larger amounts of food allergens until target dose is reached Education, training, and support for the medical community and families affected by food allergies Build-Up Phase ~6 months Ongoing Maintenance / Potential Immunomodulation Characterized Oral Desensitization ImmunoTherapy 5

6 Our Pipeline of Treatments for Food Allergies Using Aimmune s Investigational CODIT Approach Pre-IND Phase 2 Phase 3 Next Milestone AR101: Peanut Allergy Fast Track and Breakthrough Therapy Designation (Ages 4-17) PALISADE Core Efficacy and Safety Study (Ages 4-55) RAMSES Real-World Experience Study (U.S. / Canada) Completed Met Primary and Key Secondary Endpoints, Ages 4-17 BLA by YE 2018 MAA by 1H 2019 Data 2H 2018 AR101 ARTEMIS European Trial Data 1Q 2019 ARC004 PALISADE Roll-Over Study Data Cut 2H 2018 ARC005 Pediatric Study Initiate P3 2H 2018 AR101 + Adjunctive Dupilumab Initiate P2 2H 2018 AR201: Egg Allergy Initiate 2019 File IND 2018 AR301: Walnut Allergy Initiate 2019 File IND

7 Industry Leaders Share Our Vision Scientific Discovery and Innovation Pipeline Expansion Building AR101 Value Scientific Discovery and Innovation Clinical collaboration (Oct 2017) with Aimmune Regeneron/Sanofi Joint Development Committee Phase 2 of AR101 with adjunctive dupilumab expected to start in 2018 * Plan to explore sustained unresponsiveness in peanut allergy Total $145M strategic equity investment (Nov 2016) $30M participation in equity financing (Feb 2018) Two-year working collaboration (ending Nov 2018) Aimmune retains full global rights to all CODIT pipeline assets, including AR101 Building AR101 Value Leading handler, processor, and exporter of peanut and tree nuts Exclusive supply of AR101 starting material through 2034 Alliance focused on protecting people with peanut allergies Equity stake in Aimmune 7 *Regeneron will sponsor and fund the trial, with Aimmune to provide clinical supply of AR101 and food challenge materials

8 Aimmune s First CODIT Focus: Peanut Allergy

9 There Is a Large and Growing Need for Peanut Allergy Treatments Prevalence in the United States and EU5 Millions of Patients (Ages 1 to 55) 8.2M U.S. Ages 4 to M 1.4M 6.4M 1.7M 7.3M 1.8M 2.0M EU5 Ages 4 to M 1.2M 1.3M 1.4M 2015E 2020F 2025F 2030F 9 Sources: Gupta (2011, 2013), Avery (2003), Cummings (2010), Sicherer (2010), Venter C (2010), Hourihane JO (2007), Nicolaou (2010), World Bank, Aimmune internal analysis

10 Avoiding Peanut Allergens Is Difficult Peanut Allergens Can Be Hard to Spot Median Accidental One Accident Exposure Is ~ 1/2 a Peanut 1 Can Be Fatal The median estimated eliciting dose in real life was 125 mg (N=238 peanut-allergic patients) as reported by Deschildre A, et al. Clinical & Experimental Allergy; 46:

11 There Is a Significant Burden of Living with Peanut Allergy Accidents are inevitable The threat of a severe peanut allergy reaction dominates families daily lives ~40% of patients experienced anaphylaxis after already being made aware of their condition 1 83% 76% There s always that fear when you can t reported that peanut allergy makes it hard to attend social events 2 reported that peanut allergy interferes with quality of life overall 2 be with her. You don t want to roll the dice and take any chances. -Parent Aimmune proprietary market research based on anaphylaxis codes (not accounting for physician under-coding) over an average 3-year observation period; Symphony Health Patient Claims (Jan 2011-Jun 2017) 2. MyFoodAllergyTeam Survey (N=129, conducted April 2017)

12 People and families affected by peanut allergy want a medication that offers Reliable Robust Real-World Protection from allergic reactions due to accidental exposures to peanut Based on qualitative interviews with 200+ allergists, patients and physicians, quantitative surveys with >300 allergists and >400 parents in the United States and EU

13 Therapeutic Goal of AR101 Is to Protect Against Allergic Reactions to Real-Life Accidental Exposures Pivotal Phase 3 PALISADE Trial Evaluated: Ability to tolerate at least 600 mg (single dose of peanut protein, ~2 peanuts) 1 Reduced frequency and severity of symptoms 2 Efficacy outcomes after ~12 months on therapy 3 AR101 Treatment Resulted in a Significant Reduction in Frequency and Severity of Allergic Reactions During Peanut Exposure in Exit Food Challenge Successful consumption of at least a single dose of 600 mg of peanut protein with no more than mild symptoms in an exit double-blind, placebo-controlled food challenge 2. In an exit double-blind, placebo-controlled food challenge 3. ~6 months up-dosing and ~6 months maintenance

14 Primary and Key Secondary Endpoints Met in Phase 3 PALISADE Trial of AR101 for Peanut Allergy (Ages 4 17) Primary endpoint: Difference between groups = 63% (95% CI: 53,73) p< mg peanut protein (~2 peanuts) is ~5X the amount of a typical accidental exposure to peanut in the real world 1 AR101 (N=372) Placebo (N=124) Single Tolerated Dose (mg) *Responders based on Intent-to-Treat, defined as subjects who tolerated each dose level with no more than mild symptoms at the exit double-blind, placebo-controlled food challenge; p< for treatment difference >15% Jones S, et al. AAAAI 2018; Du Toit, G et al. EAACI The median estimated eliciting dose in real life was 125 mg (N=238 peanut-allergic patients) as reported by Deschildre A, et al. Clinical & Experimental Allergy; 46:

15 Longitudinal Change in Single Highest Tolerated Dose ITT Analysis: Median Amount of Peanut Protein Tolerated in Entry & Exit DBPCFCs 1000 mg 100-Fold Increase in AR101 Group 30 mg 3-Fold Increase in Placebo Group 10 mg 10 mg (N=372) (N=124) 15 ITT: Intent-to-Treat Population, 4-17 age group DBPCFCs: Double-Blind, Placebo-Controlled Food Challenges Jones S, et al. AAAAI 2018

16 Responders % Exploratory Analysis of Similarities Across Age Groups Completers: Subjects Across Age Cohorts Who Tolerated 600 mg at Exit DBPCFC % 83% 85% AR101 Placebo % 5% 3% 0 Ages 4-11 (n=281) Ages (n=131) Ages (n=33) * * On an intent-to-treat basis, the p-value was p=0.07 Single Tolerated Dose of 600 mg 16 DBPCFC: Double-Blind, Placebo-Controlled Food Challenge Responders defined as subjects who tolerated each dose level with no more than mild symptoms at the exit DBPCFC Du Toit, G et al. EAACI 2018

17 Subjects Having Symptoms (%) Subjects Having Symptoms (%) AR101 Reduced Frequency and Severity of Allergic Reactions to Peanut Exposure During Exit DBPCFC Placebo AR101 None Mild Moderate Severe Subjects Attempting Dose Subjects Attempting Dose Percent Reduction in Epinephrine Use During Exit DBPCFC (AR101 vs. Placebo) 1 mg N/A 3 mg 100% 10 mg 100% 30 mg 100% 100 mg 99% 300 mg 99% 600 mg 94% 1000 mg 81% 17 Dose level, mg DBPCFC: Double-Blind, Placebo-Controlled Food Challenge Jones S, et al. AAAAI 2018

18 Encouraging Safety Profile of AR101 Over 12 Months of Treatment (Ages 4 17) 80% of patients completed the trial Most experienced mild or moderate side effects Typically gastrointestinal symptoms during the up-dosing phase; led to discontinuation in 6.5% of subjects overall 1.9% of subjects discontinued due to systemic allergic reactions In ~112,000 doses of AR101 administered (307 patient-years of exposure), there was one case each of eosinophilic esophagitis and severe anaphylaxis (less than 1% of subjects overall) AR101 reduced the frequency of allergic reactions, symptom severity and rescue epinephrine use during the exit food challenge 18 Hourihane et al, Efficacy and safety of AR101: results of the phase 3 peanut allergy oral immunotherapy study for desensitization (PALISADE) trial, BSACI (British Society of Allergy and Clinical Immunology), Sept 30, Telford, UK

19 AR101 Commercial Launch Plans Are Focused on U.S. Allergists

20 Allergists Want an FDA-Approved Oral Immunotherapy ~5,000 Allergists in the United States ~75% Surveyed Would Prescribe an FDA-Approved Oral Immunotherapy Survey of 442 members of The American Academy of Allergy, Asthma & Immunology (AAAAI) reported by Greenhawt M and Vickery B, Allergist-Reported Trends in the Practice of Food Allergen Oral Immunotherapy. J Allergy Clin Immunol Pract (2015); 3(1):

21 Many Allergists View Food Allergy Therapeutics as Very Important to the Future of Their Practices Food-allergic patients tend to have several co-morbidities due to the Atopic (Allergic) March The Allergic March Phase 3 PALISADE (Ages 4-17) 72% history of anaphylaxis 53% asthma 66% multiple food allergies 72% allergic rhinitis Eczema Food Allergy Rhinitis Asthma AR101 and other investigational CODIT treatments may help anchor a deep long-term relationship between allergist and patient at an early age 21

22 If Approved AR101 Would Fit Well With Current Allergy Practice Additive to Practice Allergists surveyed believe their practices today have the capacity to treat patients with AR101. Basic Infrastructure Exists Practice management with AR101 should be similar to allergy shots for environmental allergies. Reimbursement Expected Physicians services for multiple office visits during up-dosing should be reimbursed as a medical benefit. 22 Based on Aimmune proprietary market research

23 Allergists Prefer Treatments that Keep Them Involved: What We Can Learn from Environmental Allergy Treatments 90% of environmental allergy patients (ages 4-17) undergoing therapy receive allergy shots 90% 10% Allergy Shots SLIT Sublingual Immunotherapy (SLIT) Grastek, Ragwitek, Oralair (examples) Requires 1-3 office visits for initial dose Patient takes subsequent doses at home Allergy Shot Immunotherapy Deemed the more effective treatment Can be personalized Requires multiple office visits for up-dosing Requires in-office waiting time after dosing Allergist stays involved in treatment AR101 Would Follow the Allergy Shots Model, Keeping the Allergist Involved in Ongoing Treatment and Patient Care 23 Source: Aimmune Market Research: Online quantitative survey (N=57 Board-certified allergists) conducted October 2017

24 AR101 Has the Potential to Capture Meaningful Market Share ~5,000 U.S. Allergists Target: 3,000 practices at peak ~1.6M U.S. Kids and Teens with Peanut Allergy Target: 150,000 up-dosed at peak >$1B Estimated U.S. Market Opportunity for AR101 with Field Team of < Estimates based on Aimmune proprietary market research

25 Strong Proprietary Position with AR101 for Peanut Allergy Biologic Data Exclusivity (12 years in the U.S.) Issued Patents Covering Formulation and Manufacturing/ Analytics Manufacturing Expertise, Trade Secrets and Know-How Exclusive Commercial Supply Agreement First Mover Advantage and Working to Build a Trusted Brand 25

26 CODIT Pipeline Drives Long-Term Value

27 Large Opportunities in Food Allergies Beyond Peanut Egg Walnut Multi-Nut Milk ~ 6M egg allergic people across US, EU5, China and Japan Majority of patients ages 1-4 years old Most common food allergy in China and Japan Accounts for up to 50% of tree nut allergies In the US, ~0.5% of children and ~1% of adults are walnut allergic Severe and unpredictable allergy; usually lifelong Incidence of multi-nut allergy is high among people who are tree nut allergic Collective prevalence approaches that of peanut allergy ~ 2.5% of children under three years old are allergic to milk Failure to thrive challenges High desire to address given milk is viewed as a nutritional requirement and very difficult to avoid 27 Estimates based on Aimmune proprietary market research

28 Event-Rich Year Ahead

29 Anticipated Milestones 2019 H1 ARTEMIS (EU) Ph3 Enrollment Complete (Mar) Additional PALISADE Ph3 data at EAACI (May) 2018 H2 Initiate AR101 + Dupilumab Trial (Regeneron/Sanofi) RAMSES Phase 3 Data ARC004 (PALISADE Follow-On) Ph3 Data cut completed Initiate AR101 Pediatric Trial (ARC005) BLA Submission for AR101 Submit IND for AR201 Egg H1 BLA submission accepted and PDUFA date set ARTEMIS Ph3 Data MAA Submission for AR101 Initiate AR201 Ph2 H2 Potential Ad Comm for AR101 Potential U.S. Approval of AR101 Potential U.S. Commercial Launch IND Submission for AR301 Walnut Initiate AR301 Walnut Ph2 Trial With $296M, We Are Well Capitalized to Deliver on Our Goals $296M Cash, cash equivalents and investments as of 06/30/18

30 Aimmune Therapeutics Emerging Leader in Food Allergy Therapeutics Innovation CODIT Creating a new treatment paradigm in food allergy AR101 Planning a 2019 U.S. commercial launch with potentially the first approved prescription medication for peanut allergy Leading the way in a new field of medicine through clinical research and strategic collaborations CODIT : Characterized Oral Desensitization ImmunoTherapy 30

31 We Invite You to Learn More