Analyst and Investor Event. March 4, 2018

Transcription

1 Analyst and Investor Event March 4, 2018

2 Welcome Laura Hansen, PhD VP, Investor Relations, Aimmune

3 Today s Agenda Time Topic Presenter 6:30 p.m. Welcome Laura Hansen, PhD VP, Investor Relations, Aimmune 6:35 p.m. Phase 3 PALISADE Results Stephen Dilly, MBBS, PhD Chief Executive Officer, Aimmune 7:05 p.m. Preparing for Commercialization of AR101 Jeff Knapp Chief Operating Officer, Aimmune 7:30 p.m. Q&A with Allergy & Immunology Expert Speaker Panel Mary Rozenman, PhD (Moderator) SVP, Corporate Development and Strategy, Aimmune 7:55 p.m. Looking Ahead Mary Rozenman, PhD SVP, Corporate Development and Strategy, Aimmune 3

4 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-K filed on February 20, 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. 4

5 PALISADE Phase 3 Trial Results Stephen Dilly, MBBS, PhD Chief Executive Officer, Aimmune

6 Phase 3 PALISADE Trial of AR101 for Peanut Allergy Met Primary and Key Secondary Efficacy Endpoints for Ages 4-17 Intent-to-Treat Efficacy: Percent of Patients Tolerating Each Dose in Exit DBPCFC Primary Endpoint 300 mg 600 mg 1000 mg AR101 (n=372) 76.6% 67.2% 50.3% Placebo (n=124) 8.1% 4.0% 2.4% 95% CI Difference ( %) ( %) ( %) p-value p< p< p< Completer Population: Percent of Patients Tolerating Each Dose in Exit DBPCFC 300 mg 600 mg 1000 mg AR101 (n=296) 96.3% 84.5% 63.2% Placebo (n=116) 8.6% 4.3% 2.6% 95% CI Difference ( %) ( %) ( %) p-value p< p< p< Primary and Key Secondary Efficacy Analyses Completed in Accordance with Hierarchical Statistical Analysis Plan in Patients Ages 4-17

7 Longitudinal Change in Highest Tolerated Dose: ITT Population Median amount of peanut protein tolerated in Entry and Exit Peanut Challenges 7 ITT: Intent-to-Treat Population, 4-17 age group

8 AR101 Placebo P a t i e n t s H a v i n g S y m p t o m ( % ) P a t i e n t s H a v i n g S y m p t o m ( % ) P a t i e n t s A t t e m p t i n g D o s e P a t i e n t s A t t e m p t i n g D o s e Symptom Severity at Exit Peanut Challenge Completers Secondary Efficacy Endpoint Epinephrine Use AR101 Placebo None 268 (91%) 54 (47%) 1 25 (8%) 43 (37%) * 3 (1%) 17 (15%) (2%) p< for overall between-group difference None Mild Moderate Severe * 2 epinephrine uses in 1 pt at 600 mg and 2 pts at 1000 mg in the AR101 arm Key Findings Compared to placebo, the AR101 group: 1. Developed fewer moderate and severe symptoms; Required more peanut exposure for the onset of symptoms; Was more likely to complete the challenge; 4. Needed less epinephrine

9 One Patient s Experience 9 Credit: Mommy2TwoBoys (YouTube)

10 Phase 3 PALISADE Trial: Study Design Up-Dosing ~6 Months Maintenance ~6 Months AR101 N=372 3 mg 300 mg Entry DBPCFC Tolerate 30 mg 3:1 Dose escalations in allergist office every 2 weeks Daily dose at home Daily 300 mg dose at home Exit DBPCFC Tolerate 600 mg Placebo N=124 Total treatment duration ~12 Months 10 PALISADE = Peanut ALlergy Oral Immunotherapy Study of AR101 for DEsensitization in Children and Adults Tolerate = dose is successfully ingested with no more than mild symptoms

11 Phase 3 PALISADE Trial: Patient Population Patients were highly atopic and highly sensitive to peanut At study entry, before treatment 72% had history of anaphylaxis 53% had asthma 66% had multiple food allergies 43% had peanut-specific IgE 100 ku/l Most patients had allergic reactions consistent with 1/10 of a peanut or less The median tolerated dose was 10 mg in the entry food challenge (~ 1/30 of a peanut) 90% of patients enrolled were ages 4-17; focus of primary analysis 11

12 AR101 Met the Primary and Key Secondary Efficacy Endpoints in Patients Ages 4-17 in the Phase 3 PALISADE Trial ITT Analysis: Patients Ages 4-17 Who Tolerated Each Dose Level at Exit DBPCFC *p< for Treatment Difference >15% 12 ITT: Intent-to-Treat

13 Phase 3 PALISADE Trial: Completer Analysis Completers: Patients Ages 4-17 Who Tolerated Each Dose Level at Exit DBPCFC *p< for Treatment Difference >15% 13 Completers: 79.6% of AR101-treated and 93.5% of placebo-treated patients were evaluable in the exit DBPCFC

14 Phase 3 PALISADE Trial: Safety and Tolerability Profile of AR patients ages 4-17 evaluable for safety (372 AR101, 124 placebo) No deaths or suspected, unexpected serious adverse reactions (SUSARs) 9 SAEs in 8 AR101 participants (2.2%) and 1 SAE in 1 placebo participant (0.8%) In the AR101-treated participants: 5 events were unrelated to study drug; 4 were related 5 events led to discontinuation 1 event was severe and related - anaphylaxis early in maintenance; high baseline psige 1 AR101 patient was diagnosed with EoE and withdrew, with resolution of symptoms 85% of AR101-treated patients did not experience investigator-reported systemic hypersensitivity reactions For the 14.5% of patients who did, 98.2% had mild or moderate reactions, and one severe (related SAE as noted above) 14 SAEs: Serious Adverse Events EoE: Eosinophilic Esophagitis

15 ~80% of AR101-Treated Patients Ages 4-17 Completed PALISADE AR101 (n=372) % n Total discontinuations regardless of causality 20.4% 1 76 Discontinuations not related to adverse events 8.0% 30 Discontinuations related to adverse events 12.4% 46 Gastrointestinal 2 6.7% 25 Systemic hypersensitivity reactions 3 2.7% 10 Respiratory system 1.1% 4 Cutaneous 0.8% 3 Other 4 1.1% 4 1 Of these, 16.7% discontinued during up-dosing and 3.8% during maintenance 2 One patient discontinued due to biopsy-confirmed moderate, non-serious eosinophilic esophagitis (EoE) during the study; symptoms had resolved by the time the patient left the study. No additional cases of EoE were identified in the study 3 Of these, 7 were investigator-identified anaphylaxis events (6 mild or moderate, 1 severe) 15 4 Includes one discontinuation for each: acute viral illness, eye pruritus, headache, and an unknown factor

16 M e a n w h e a l d i a m e t e r a b o v e n e g c o n t r o l ( m m ) k U A / L I g E / I g G 4 R a t i o AR101 Treatment Associated with Marked Immune Modulation I g E I g E / I g G 4 r a t i o A R A R P l a c e b o P l a c e b o 8 0 p=0.5* p<0.0001* Key Findings B a s e l i n e E n d o f U p d o s i n g E x i t Compared to placebo, the AR101 group had: 1. A reduction in psige 2. A reduction in SPT 3. A reduction in psige/igg4 ratio due to an increase in IgG S P T 0 B a s e l i n e E n d o f U p d o s i n g E x i t p<0.0001* A R P l a c e b o *p value refers to a between-group comparison of the change from Baseline to Exit using an ANCOVA model 0 16 SPT: Skin Prick Test B a s e l i n e E n d o f U p d o s i n g E x i t

17 Exploring Association of psige Levels with Patient Experience on AR101 Analysis of 4-17 y.o. Patient Outcomes, N (%) Intent-to-Treat Population PS IgE 100 ku/l N=213 PS IgE > 100 ku/l N=159 PS IgE 100 ku/l N=176 PS IgE > 100 ku/l N=120 Tolerated 1000 mg* 117 (55%) 70 (44%) 117 (67%) 70 (58%) Discontinued due to GI AEs 10 (5%) 15 (9%) 0 0 Experienced a severe SHR 0 1 (0.6%) 0 0 * Key secondary endpoint Compared to patients with psige > 100 ku/l, patients with peanut-specific IgE 100 ku/l after one year on AR101 therapy: Had a higher overall completer rate (83% vs. 75%) A higher response rate at the 1000 mg endpoint (55% vs. 44%) Fewer GI related withdrawals (5% vs. 9%) No severe treatment-related adverse events (0 vs. 1 event) Completer Population 17 GI AEs: Gastrointestinal Adverse Events SRH: Investigator-reported Systemic-Hypersensitivity Reaction

18 Responders % Exploratory Analysis of Similarities Across Age Groups Completers: Patients Across Age Cohorts Who Tolerated 600 mg at Exit DBPCFC % 82.8% 85.0% AR101 Placebo (n=197) (n=99) (n=20) % 3.1% (n=84) (n=32) Ages 4-11 (n=281) 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=

19 Phase 3 PALISADE Trial: Key Takeaways and Implications Met primary and secondary efficacy endpoints Strong efficacy and encouraging safety profile Provides core element of our Biologics License Agreement (BLA) filing Moving ahead to complete RAMSES real-world trial in support of safety database requirement Poised to deliver potentially the first treatment for peanut allergy 19

20 AR101 Clinical Path to Regulatory Submissions PALISADE core pivotal Phase 3 trial RAMSES trial (U.S./Canada) Real-world safety and tolerability trial Data expected in 2H 2018 ARC004 (PALISADE follow-on trial) Data-cut expected in 3Q 2018 AR101 has U.S. FDA Breakthrough Therapy Designation for peanut-allergic patients ages 4 17 Clinical Trials Supporting BLA Submission by Year-End 2018 PALISADE core pivotal Phase 3 trial ARTEMIS trial (Europe) Complete study by YE 2018 Data expected in 1H 2019 ARC004 (PALISADE follow-on trial) Data-cut expected in 3Q 2018 ARC005 Pediatric trial First-patient-in fulfills MAA requirement Initiate 2H 2018 Clinical Trials Supporting MAA Submission in 1H

21 Phase 3 PALISADE Trial of AR101 for Peanut Allergy Met Primary and Key Secondary Efficacy Endpoints for Ages 4-17 Intent-to-Treat Efficacy: Percent of Patients Tolerating Each Dose in Exit DBPCFC Primary Endpoint 300 mg 600 mg 1000 mg AR101 (n=372) 76.6% 67.2% 50.3% Placebo (n=124) 8.1% 4.0% 2.4% 95% CI Difference ( %) ( %) ( %) p-value p< p< p< Completer Population: Percent of Patients Tolerating Each Dose in Exit DBPCFC 300 mg 600 mg 1000 mg AR101 (n=296) 96.3% 84.5% 63.2% Placebo (n=116) 8.6% 4.3% 2.6% 95% CI Difference ( %) ( %) ( %) p-value p< p< p< Primary and Key Secondary Efficacy Analyses Completed in Accordance with Hierarchical Statistical Analysis Plan in Patients Ages 4-17

22 Preparing for Potential Commercialization of AR101 Jeff Knapp Chief Operating Officer, Aimmune

23 Market Research Shows that Patients Want a Bite-Proof Level of Protection They Can Count On Protection from Accidental Exposure Bite-Proof * No or Reduced Reactions Certainty Rapid Effect Therapeutic goal of AR101 aligns with what patients want in a peanut allergy therapy 23 Based on qualitative interviews with 200+ allergists, patients and physicians, quantitative surveys with >300 allergists and >400 parents in U.S. and EU, ongoing input from Aimmune SAB and KOL community *2 peanuts is ~600 mg (e.g., child s bite of peanut butter sandwich)

24 Of the 1.6M U.S. Peanut Allergic Patients, AR101 Has the Potential to Capture Meaningful Market Share 30% Diagnosed and Managed by Pediatricians (top 4% manage 40% of patients) 70% Diagnosed and/or Managed by Allergists Aimmune s target audience Potential Future Referrals to Allergists AR101 Would Fit Well Into Current Allergy Practice 1.6 Million Potential Patients Ages 4-17 ~1 million clinically managed today Expected Drivers of Patient Motivation to Seek AR101 Therapy Reaction History Including Proximity of Events Childhood Milestones Anxiety Family Support 24 Source: Symphony claims data,

25 High Unmet Need for Desensitization to Peanuts Based on U.S. Patient Claims Data 1M Total Diagnosed Peanut Allergy (PA) Patients (Age 4 to 17) 1M First Experience With PA Can Be Very Traumatic Almost one in five* diagnosed PA pts learned about their condition via an anaphylactic event that resulted in a trip to the ER Accidental Exposure Resulting In ER or HCP Visit About one-third* of pts experienced anaphylaxis after they had already been made aware of their condition 17.5% 175K 30% 300K 25 *Average 3-year observation period Source: Symphony Health Patient Claims (Jan 2011-Jun 2017); anaphylaxis codes: , T78.01XX, L50, L50.1, L50.3 L50.8, L50.9, T78.3, 708.0, 708.1, 708.3, 708.8, 708.9, 995.1

26 AR101 Dosing Would Fit Well Into Allergy Practices ~5,000 U.S. Allergists U.S. Allergists are highly motivated to offer FDA approved treatments AR101 in-office updosing would be similar to allergy shots Practice capacity currently exists to integrate and scale AR101 ~75% of U.S. allergists surveyed by AAAAI would prescribe an FDA-approved oral immunotherapy* Expect group practices to be high adopters; will put pressure on others to avoid losing patients Insights based on Aimmune-sponsored market research with U.S. allergists and practice managers (N=70) 26 *Greenhawt, Vickery, JACI (2015) Survey of members of The American Academy of Allergy, Asthma & Immunology (AAAAI)

27 AR101 Has the Potential to Create a New Treatment Paradigm for Peanut Allergy Unmet Need Lack of treatment options High risk of potentially life-threatening reaction High impact on parent / patient quality of life Likelihood to Prescribe Likelihood to prescribe AR101 was 6.1 on a scale of 1 to 7 (n=30) Value proposition based on Phase 2 data compelling, primarily as a safety net against severe reactions due to accidental exposure 27 Homebrew OIT Pain Points Sources: Aimmune Market Research 2017 Allergists offering homebrew OIT today believe, if approved, AR101 will address most of their pain points The need for an FDA approved product The need for a standardized product and a well tested, consistent protocol Limited and unpredictable reimbursement The need for scalability

28 Allergists are the Ideal Specialty to Own AR Patients with food allergies are generally followed by an allergist 2. Allergists have resources and training to optimally treat food allergic patients 3. Allergists have a strong desire to treat their patients with food allergies 4. Allergists are losing some patient populations to other specialties Patients will call us once the word is out. We won t have to call the patients. These patients only come in once a year now. They will have more office visits with this program. Also, I may attract new patients if I offer this. If I m not offering it, I m at a disadvantage as other Allergists will. 28 Source: Aimmune Physician and Practice Manager Market Research 2017

29 If Approved, AR101 Can Be Integrated into Allergy Offices with Existing Available Capacity and Will Be Further Optimized Over Time Seamless Integration Additive: Allergists surveyed believe their practices today have the capacity to meet the future unmet need of peanut allergic patients seeking AR101 Logistics: If approved, practice management with AR101 should be similar to allergy shots, which represents ~30% of their business High Patient Capacity If approved, Aimmune will initially target larger group Allergy practices, representing ~70% of offices At peak 3,000 unique Allergy offices each able to up-dose ~50 patients per year = 150,000 patients New Patient Starts per 6-Month Period 3 patients per day 4 days per week for 2 weeks 24 patients total Repeat 6 months later ~50 patients per year 29 Sources: Aimmune Market Research 2017

30 Access to AR101 Throughout Treatment is Intended to be Seamless to Support a Positive Patient Experience Peanut Allergy Diagnoses & Treatment Decision Initial Day Escalation (in office) Day 1 Level 1 (in office supply) Days 2-14 Level 1 (at home specialty pharmacy delivery) Maintenance 30 Day Supply (at home) Repeat through Level 11 AR101 Patient Support Services Initiation Support Benefits investigation PA processed Financial counseling Starter program Ongoing Support Logistics coordination with patient and office Refill reminders and messaging Adherence and compliance support Out-of-pocket support 30

31 CPT Reimbursement Codes: Current Use for Home-Brew and Potential Future Simplification for AR101 Today: Existing codes provide adequate reimbursement according to some home-brew OIT offices Home-brew OIT Initial Diagnosis Standard office visit code (992xx) Up-dosing involves a combination of one or more allergy immunotherapy codes: Rapid desensitization code (95180) Oral food challenge codes (95076) Standard office visit code (992xx) Potential Future: New code(s) may simplify the AR101 learning curve for Allergists and Payers AR101 Initial Diagnosis Standard office visit code (992xx) Up-dosing could be reduced to one time bound code Requires support from societies (ACAAI & AAAAI) 31 CPT: Current Procedural Terminology Source: Aimmune Physician and Practice Manager Market Research 2017

32 U.S. Payer Reimbursement Outlook for AR101 Appears Favorable* Payer Management of Peanut Allergy Expected to be Low Payers Likely to Cover AR101 Given Promising Value and High Unmet Need Payers Expected to Require a Standard Prior Auth. for AR101 ~90% of payers surveyed exert low or no effort in managing food and peanut allergy today Management not expected to increase significantly in near term given low spend vs. other therapeutic areas In recent ad board, 10/10 payers indicated they expect to add AR101, if approved, to formulary (tier variable) based on: Promising clinical value Life threatening pediatric indication Lack of alternatives In blinded research, 90% of payers expect to require a standard annual Prior Authorization (PA) for AR101 PA criteria may include treatment by an allergist and confirmed documented diagnosis of peanut allergy 32 *Sources: Payer Online Market Research (n=30), Aug 2017; Payer Advisory Board (n=10), Oct 2017

33 Summary - Commercial Outlook for AR101 Very large peanut allergic population with no existing treatment options Majority of existing peanut allergic patients are seen by Allergists Allergists are well equipped to treat peanut allergic patients with AR101 High likelihood to prescribe AR101 based on existing data/profile Allergists are eager to own something other specialties are not equipped to utilize Allergists have capacity to incorporate AR101 patients. This will be an additive source of patient volume Administration of allergy shots is similar to the OIT process & the adoption of AR101 is a natural progression Aimmune plans to be ready to execute a successful launch! 33

34 Q&A with Allergy & Immunology Experts Speaker Panel Moderator: Mary Rozenman, PhD SVP, Corporate Development & Strategy, Aimmune

35 Allergy & Immunology Experts Speaking Today Daniel Adelman, MD Christina Ciaccio, MD Edwin Kim, MD Ellen Sher, MD Stephen Tilles, MD UCSF Aimmune Therapeutics University of Chicago School of Medicine University of North Carolina Chapel Hill Allergy Partners of New Jersey University of Washington Northwest Asthma and Allergy Center Disclosures: Disclosures: Disclosures: Disclosures: Disclosures: Aimmune Employee Clinical Research Principal Investigator: Aimmune Clinical Research Principal Investigator: Aimmune, Astellas, DBV, HAL Allergy Consultant for Aimmune, DBV Clinical Research Principal Investigator: Aimmune Consultant for Aimmune, ALK, Optinose Consultant: Aimmune, Sanofi, Before Brands, DOTS Technology, DBV Clinical Research Principal Investigator: Aimmune, AnaptysBio, Astellas, DBV, Sanofi 35

36 Looking Ahead Mary Rozenman, PhD SVP, Corporate Development & Strategy, Aimmune

37 Collaborations Focused on Advancing the Field of Food Allergy Leading handler, processor, and exporter of peanut and tree nuts Exclusive supply of AR101 starting material through 2033 Alliance focused on protecting people with peanut allergies Equity stake in AIMT $145M strategic equity investment (Nov 2016) Two-year working collaboration Aimmune retains full global rights to all CODIT pipeline assets, including AR101 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 Building AR101 Value Pipeline Expansion Scientific Discovery and Innovation 37 *Regeneron will sponsor the trial, with Aimmune to provide clinical supply of AR101 and food challenge materials

38 Anticipated Milestones Milestones Late-Breaker on PALISADE Ph3 at AAAAI ARTEMIS (EU) Ph3 Enrollment Complete RAMSES (Real-World) Ph3 Data ARC004 (PALISADE Follow- On) Ph3 Data Cut BLA Submission for AR101 Initiate AR101 Pediatric Trial (ARC005) Initiate AR101 + Dupilumab Ph2 Trial (Regeneron/Sanofi) IND Submission for Egg Allergy ARTEMIS (EU) Ph3 Data MAA Submission for AR101 Potential Approval of AR101 IND Submission for Walnut Allergy Initiate Egg and Walnut Ph2 Trials With ~$372 M in Cash * We Are Well Capitalized to Deliver on Our Mission 38 *$182.4M cash, cash equivalents and investments as of 12/31/17 + $190M net proceeds from recent financing

39 Thank You