Corporate Presentation. June 2018

Transcription

1 Corporate Presentation June 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 May 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: The Food Allergy Therapeutics Company Our Urgent Priority Provide kids, young adults and their families protection, peace of mind, and freedom from food allergies. 3

4 There Is a Large and Growing Need for Food Allergy Treatments Responsible for 200,000 visits to U.S. emergency rooms each year 1 Cost American families ~ $25 billion a year, with ~ $4 billion attributed to direct medical expenses 2 Living with food allergies means living with constant risk and uncertainty Food allergies can lead to life-threatening reactions There are no approved treatments for food allergies >30M 4 1. Clark S et al. J Allergy Clin Immunol. 2011;127(3): Gupta R, et al. JAMA Pediatr. 2013;167(11): National Institute of Allergy and Infectious Diseases, National Institutes of Health. Retrieved from 4. United States Census Bureau Quick Facts (2015 estimates). 5. Gupta RS, et al. Pediatrics 2011;128(1):e EAACI. Food Allergy & Anaphylaxis Public Declaration. People have food allergies in the United States and Europe 3-6

5 Aimmune was founded to address the collective call to action from food allergy leaders and patient advocates for the development of an FDA-approved biologic oral immunotherapy (OIT) for the treatment of food allergies. 5

6 Aimmune Is Developing the CODIT Approach to Treat Food Allergies Characterized Oral Desensitization ImmunoTherapy A standardized oral immunotherapy (OIT) protocol with precise oral dosing FDA-regulated biologic products Training and education Goal: Gradually increase the body s ability to tolerate larger amounts of allergenic food proteins without mounting an allergic reaction. ~12 visits to the allergist twice a month for 6 months and daily at-home dosing between visits Convenient daily dose taken with food 6

7 Our Pipeline of CODIT Treatments for Food Allergies Biologic Drug Products and Protocols 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 Endpoint, 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 *Primary Endpoint: The proportion of patients who could tolerate at least 600 mg of peanut protein (~2 peanuts) in an exit double-blind, placebo-controlled food challenge

8 Industry Leaders Share Our Vision Scientific Discovery and Innovation Pipeline Expansion Building AR101 Value 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 $145M strategic equity investment (Nov 2016) $30M participation in equity financing (Feb 2018) Two-year working collaboration Aimmune retains full global rights to all CODIT pipeline assets, including AR101 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 AIMT 8 *Regeneron will sponsor the trial, with Aimmune to provide clinical supply of AR101 and food challenge materials

9 Aimmune s First CODIT Focus: Peanut Allergy

10 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 10 Sources: Gupta (2011, 2013), Avery (2003), Cummings (2010), Sicherer (2010), Venter C (2010), Hourihane JO (2007), Nicolaou (2010), World Bank, Aimmune internal analysis

11 Avoiding Peanut Protein Is Very Difficult Peanut Protein Can Be Difficult to Detect People With Peanut Allergies Can Be Sensitive to the Amount of Peanut Protein Found in a Fraction of a Peanut Kernel 1,2 One Accident Can Be Fatal Allen K, et al. Allergen reference doses for precautionary labeling (VITAL2.0): Clinical implications. J Allergy Clin Immunol. 2014; 133(1): Deschildre A, et al. Peanut-allergic patients in the MIRABEL survey: characteristics, allergists dietary advice and lessons from real life. Clinical & Experimental Allergy; 46:

12 There Is a Significant Burden of Living with Peanut Allergy Constant Risk and Uncertainty Due to Likelihood of Reaction to Accidental Exposure Peanut Allergy Can Substantially Diminish Quality of Life of Patients and Their Families ~40% of Patients Experienced Anaphylaxis After Already Being Made Aware of Their Condition 1 83% 76% There s always that Reported that Peanut Allergy Makes it Hard to Attend Social Events 2 Reported that Peanut Allergy Interferes with Quality of Life Overall 2 fear when you can t be with her. You don t want to roll the dice and take any chances. -Caregiver 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)

13 Therapeutic Goals of AR101 Align With What Patients Want Bite-Proof level protection e.g., a child s bite of a peanut butter sandwich (~2 peanuts) No or reduced reactions Certainty Market Research 1 Shows Parents and Patients Want: We Designed PALISADE Phase 3 to Evaluate: Ability to tolerate at least 600 mg (~2 peanuts) 2 Reduced symptom severity Daily oral maintenance dosing with AR101 Rapid effect Efficacy outcomes after ~12 months on therapy Based on qualitative interviews with 200+ allergists, patients and physicians, quantitative surveys with >300 allergists and >400 parents in the United States and EU 2. Defined as successful consumption of at least 600 mg peanut protein with no more than mild symptoms in an exit double-blind, placebo-controlled food challenge 3. ~6 months up-dosing and ~6 months maintenance

14 Phase 3 PALISADE Results (Ages 4 17) Primary and Key Secondary Efficacy Endpoints Met 600 mg peanut protein is approximately 2 peanuts Primary endpoint: Difference between groups = 63% (95% CI: 53,73) p< Safety Over 12 Months of Treatment More than 111,000 doses of AR101 administered (307 patient-years of exposure) No suspected, unexpected serious adverse reactions No life-threatening adverse events 4 (1.1%) subjects experienced treatment-related serious adverse events One case of severe anaphylaxis (subject withdrew from the trial) 6.7% gastrointestinal adverse events that led to discontinuation AR101 (n=372) Placebo (n=124) One case of eosinophilic esophagitis (resolved after study discontinuation) *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% 14 Jones S, et al. AAAAI 2018

15 Longitudinal Change in Highest Tolerated Dose ITT Analysis: Median Amount of Peanut Protein Tolerated in Entry & Exit DBPCFCs 1000 mg 10 mg 10 mg 30 mg 15 ITT: Intent-to-Treat Population, 4-17 age group DBPCFCs: Double-Blind, Placebo-Controlled Food Challenges Jones S, et al. AAAAI 2018

16 Subjects Having Symptoms (%) Subjects Having Symptoms (%) AR101 Symptom Severity at Exit DBPCFC Placebo AR101 Subjects Attempting Dose Subjects Attempting Dose Symptom Severity Scoring Key None Mild Moderate Severe Compared to placebo, the AR101 group developed fewer moderate and severe symptoms 16 DBPCFC: Double-Blind, Placebo-Controlled Food Challenge Jones S, et al. AAAAI 2018

17 Responders % Exploratory Analysis of Similarities Across Age Groups Completers: Subjects Across Age Cohorts Who Tolerated 600 mg at Exit DBPCFC % 82.8% 85.0% AR101 Placebo % 3.1% (n=84) Ages 4-11 (n=281) (n=197) (n=99) (n=20) (n=32) Ages (n=131) 15.4% (n=13) Single Highest Tolerated Dose of 600 mg Ages (n=33) * * On an intent-to-treat basis, the p-value was p= 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

18 Efficacy Across All Ages Is Important as Peanut Allergy Does Not Usually Resolve With Age 1 Ages Estimated Percent of Total Peanut- Allergic Patients % 27% 10% 46% PALISADE Phase 3 trial primary endpoint analysis conducted in 4-17 age group 2. Estimates based on Aimmune proprietary market research

19 Potential AR101 Commercial Launch to Focus on U.S. Allergists

20 Allergists Are Ready for 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 Viability 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 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. 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 Community Allergists Will Drive AR101 Adoption, If Approved Importance to Practice Dynamics Reimbursement for Services Matter Clinic Time Is Valuable Allergy Shot Experience Is Leveraged Excited for a New Patient Population Practice Settings Academia Community Practice 23 Based on Aimmune proprietary market research

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 Sales Force 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 Exclusive Commercial Supply Agreement Manufacturing Expertise, Trade Secrets and Know-How First Mover Advantage and Building a Trusted Brand 25

26 CODIT Pipeline Drives Long-Term Value

27 Large Opportunities in Food Allergies Beyond Peanut Egg Walnut Multi-Nut Milk In 2025 there may be ~ 1.7M egg allergic patients across US, EU5, China and Japan Majority of patients ages 1-4 years old Most common food allergy in China and Japan High prevalence that has significantly increased in the last ~20 years 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 H1 $332M We Are Well Capitalized to Deliver on Our Goals 1 ARTEMIS (EU) Ph3 Enrollment Complete (Mar) Additional PALISADE Ph3 data at EAACI (May) 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 Initiate AR201 Ph2 MAA Submission for AR101 IND Submission for AR301 Walnut H2 Potential Ad Comm for AR101 Potential U.S. Approval of AR101 Potential US Commercial Launch Initiate AR301 Walnut Ph2 Trial $332M Cash, cash equivalents and investments as of 03/31/

30 Aimmune Is Leading the Quest for Food Allergy Therapeutics Patient-Focused A treatment paradigm in food allergy Potentially the first FDA-approved medicine for peanut allergy Driving the development of new medicines for food allergy CODIT AR101 Innovation 30

31 We Invite You to Learn More

32 Aimmune Executive Team Leader Role Experience Stephen Dilly, MBBS, PhD* Chief Executive Officer Daniel Adelman, MD Chief Medical Officer Sue Barrowcliffe Eric Bjerkholt Jeffrey Knapp* Mary Rozenman, PhD General Manager of Europe Chief Financial Officer Chief Operating Officer SVP, Corporate Development and Strategy Experienced Team of Drug Developers with 30+ NDAs, BLAs and MAAs Doug Sheehy General Counsel and Secretary William Turner SVP, Global Regulatory and Quality 32 Dr. Jayson Dallas will assume the role of President and CEO on June 19, 2018, and Dr. Stephen Dilly will become Special Advisor to Aimmune through 2018; Jeff Knapp will depart Aimmune on July 6, 2018.

33 Aimmune Top Shareholders 33 FIRM NAME % OS POSITION Nestle Health Science US Holdings, Inc ,489,584 Longitude Capital Management Co., LLC ,013,134 T. Rowe Price Associates, Inc ,492,077 Foresite Capital Management, LLC ,254,965 Fidelity Management & Research Company ,615,171 Aisling Capital, LLC ,240,000 Wellington Management Company, LLP ,061,192 Eventide Asset Management, LLC ,564,983 Eagle Asset Management, Inc ,400,482 Palo Alto Investors LP ,356,514 AllianceBernstein L.P ,331,941 Point72 Asset Management, L.P ,079,900 Explore Holdings, L.L.C ,024,117 Great Point Partners, LLC ,579 Emerald Advisers, Inc ,471 Balyasny Asset Management LP ,875 Deerfield Management Company, L.P ,210 Baker Bros. Advisors LP ,908 Victory Capital Management Inc ,641 Morgan Stanley Wealth Management ,634 TLP Trading, LLC ,000 Adage Capital Management, L.P ,000 Franklin Advisers, Inc ,900 Loomis, Sayles & Company, L.P ,320 Dilly (Stephen George Ph.D.) ,008 Jennison Associates LLC ,866 Vivo Capital, LLC ,872