Corporate Presentation Less risk. More life - November 2017 - Jefferies
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Corporate Presentation November 2017
Less risk. More life.
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 November 6, 2017.
Copies of this filing are available online at www.sec.gov or www.aimmune.com. 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
Food Allergy:
A Serious Disease with Real Unmet Need
Allergic Response Allergy to Foods
to Environmental
Allergens
(Pollen, Ragweed,
≠ (Peanut, Tree Nut, Shellfish, Egg, Milk)
Grass, Dust Mite, etc.) Constant Risk and Uncertainty
No Approved Treatment
200,000 ER Visits/Year
Nuisance and ~200 Deaths/Year
Inconvenience
OTC Medication
Millions Seeking
No Deaths Life-Protecting Therapy
3 Source: FARE, FAAN
Critical Unmet Need in Peanut Allergy and Beyond
Large and Growing Peanut Allergy Need Many Other Prevalent Food Allergies
Prevalence in U.S. and EU5 Prevalence within
Millions of patients (ages 1 to 55) Food-Allergic Population*
8.2M
7.3M Peanut 25.0%
6.4M 2.0M
Milk 21.1%
1.8M
5.4M
1.7M Shellfish 17.2%
1.4M
Tree Nut 13.1%
U.S.
Egg 9.8%
1.4M
1.3M
1.2M Fin Fish
1.1M 6.2%
EU5
Wheat 5.0%
2015E 2020F 2025F 2030F Soy 4.6%
U.S. ages 4 to 17 EU5 ages 4 to 17
*Children under age 18 years
4 Sources: FARE, Gupta (2011, 2013), Avery (2003), Cummings (2010), Sicherer (2010), Venter C (2010), Hourihane JO (2007), Nicolaou (2010), World Bank, Aimmune internal analysis
Aimmune’s Approach to Treat Food Allergy: CODIT™
Treatment Protocol
Up-Dosing Phase
Ongoing Maintenance
~6 Months
100X
Characterized mg
Oral
Desensitization X
2 weeks at each step
mg
ImmunoTherapy ~11 visits to allergist office
5
AR101: Investigational Treatment for Peanut Allergy
• Oral biologic immunotherapy; first CODIT™ application
• Breakthrough Therapy Designation for peanut-allergic
patients 4-17 years old
• Robust Phase 3 program in 1,000+ patients in North
America and Europe
• Core Phase 3 PALISADE trial fully enrolled (N=554);
final study visits expected around year-end ’17; topline
data expected in 1Q 2018
6
Understanding the Clinical Trial Endpoint: Tolerated Dose
Double-Blind, Placebo-Controlled Food Challenge (DBPCFC)
Average Accidental Exposure Level
Triggering an Allergic Reaction* • DBPCFC is done at trial
entry and exit
ENTRY
Tolerate 3 10 30 100 300 600 1,000 Single • DBPCFC patients are given
Dose
≤ 30 mg increasing doses of peanut
React React React React
protein every 20-30
Milligrams of Peanut Protein minutes to measure their
EXIT tolerated and reactive
Single
Tolerate 3 10 30 100 300 600 1,000 Dose levels
≥ 600 mg
• Given dose level is
tolerated if patient
Endpoints in Phase 2 and Phase 3 studies successfully ingests it with
PALISADE FDA primary endpoint is tolerating 600 mg
no dose-limiting symptoms
(EMA primary is tolerating 1,000 mg)
7 * Deschildre A, et al. (2015) Clinical & Experimental Allergy, 46, 610-620
AR101 Phase 2 Data: Level of Protection at End of Up-Dosing
Percentage of Subjects Tolerating Specific Test Dose Levels at 6-Month Exit Food Challenge
300 mg = 1 peanut
600 mg = child’s bite of peanut butter sandwich
Intent-to-Treat (ITT) Completers
N=29 active, N=26 placebo N=23 active, N=26 placebo
Difference between p < 0.0001
100 groups = 60% 100
p
ARC001 Phase 2 Double-Blind, Placebo-Controlled Trial: Summary*
• N=55 patients, ages 4-21 years, randomized 1:1 to daily AR101 (N=29) or placebo (N=26) and
gradually up-dosed from 0.5 to 300 mg/day
• Included patients who tolerated no more than 30 mg peanut protein at entry DBPCFC
• Primary endpoint was the proportion of subjects in each arm who tolerated a single highest
dose of at least 300 mg peanut protein in the exit DBPCFC with no or mild symptoms;
secondary endpoint, 600 mg
• On an Intent to Treat (ITT) basis, the difference in response rate between treated and placebo
subjects at the 6-month DBPCFC was
– 60% (p
AR101 Phase 2 Data: Reduced Symptom Severity
6-Month Exit Food Challenge*
N=26 N=25 N=23 N=17 N=12 N=6
100%
Placebo 50%
Maximal
0% Symptom
Single Dose: 3 mg 10 mg 30 mg 100 mg 300 mg 600 mg Severity
Severe
N=44 N=44 N=44 N=44 N=44 N=42 Moderate
100% Mild
None
AR101 50%
0%
Single Dose: 3 mg 10 mg 30 mg 100 mg 300 mg 600 mg
Real World:
*Includes placebo patients who crossed over to AR101
10 Burks W, et al. EAACI 2015; Bird A, et al. AAAAI 2016ARC002: Desensitization at the 9-Month Food Challenge
ITT* Completers
Percent of Patients Tolerating
(n=40)
(n=40) (n=36)
(n=36)
(n=24)
(n=24)
Single dose, mg 300 600 1000 300 600 1000
Cumulative dose, mg 443 1043 2043 443 1043 2043
* N= 47 comprised of n=21 ARC001 Active Rollovers + n=26 Placebo Crossovers to AR101 treatment in ARC002
11 Bird A, et al. AAAAI 2016Phase 2 Data: Continued Effect at Month 9
9-Month Exit Food Challenge*
(6 Months Up-Dosing + 3 Months Maintenance at 300 mg/day)
Exit Food Challenge Symptom Severity
Maximal Symptom Severity None Mild Moderate Severe
N=40 N=40 N=40 N=40 N=40 N=39†
100%
50%
0%
Single Dose: 3 mg 10 mg 30 mg 100 mg 300 mg 600 mg
Real World:
⃰ Includes
placebo patients who cross over to treatment with AR101
†One patient elected not to continue beyond the 300 mg food challenge step
12 Burks W, et al. EAACI 2015; Bird A, et al. AAAAI 2016AR101 Phase 2 Data: Safety and Tolerability Profile
• 80% (44/55) of patients completed AR101 up- • >90% of treatment-related AEs were mild,
dosing consistent with exposure to low levels of food
allergen (itchy mouth, hives, GI intolerance);
• 18% (10/55) withdrew due to GI AE
rest were moderate
– Onset of symptoms typically occurred within first
6 weeks and resolved within 3 weeks of AR101 • Treatment-related AE rate decreased with
cessation time on therapy
– Biopsy-confirmed EoE was seen in 1 subject – 1 AE per 30 days during up-dosing**
(1.8%)
– All had psIgE > 100 kU/L at baseline – 1 AE per 574 days during maintenance†
• 95% at-home dosing adherence* • No treatment-related withdrawals during
maintenance
Majority of Patients Completed AR101 Tolerability Improved Over Time
Up-dosing in Line with Desensitization‡
GI=Gastrointestinal; AEs=Adverse Events; psIgE=peanut-specific IgE
*Jones S, et al AAAAI 2017: ARC001 actives (N=29) and placebo (N=26); ** Bird A, et al AAAAI 2016: ARC002 placebo crossovers (N=26);
†Rachid R, et al EAACI 2016: ARC002 Part 2 low-dose extended maintenance (N=11);
13 ‡Bird A, et al AAAAI 2016: ARC002 90% of completers tolerated >600 mg; 60% of completers tolerated >1,000 mg after 9 months (n=40)PALISADE: Core Phase 3 Efficacy and Safety Trial of AR101
• 554 patients ages 4-49 enrolled (U.S., CAN,
EU); Up-dosing complete, final study visits Up-Dosing Maintenance
expected around year-end 2017 ~6 Months ~6 Months
• Included patients who tolerated ≤ 30 mg AR101
3 300
peanut protein at entry mg mg
• Daily dose at • Daily 300 mg
• Primary efficacy analysis in 4-17 age group home dose at home
Entry 3:1 Exit
(90% of enrolled patients); adults analyzed • Dose
DBPCFC escalations in DBPCFC
separately
allergist office
Tolerate Tolerate
every 2 weeks
• FDA primary efficacy endpoint is tolerating a ≤ 30 mg ≥ 600 mg
single highest dose of at least 600 mg in the
Placebo
exit food challenge (1,000 mg for EMA
analysis); lower bound of the two-sided 95%
confidence interval of the difference between Total treatment duration
AR101 and placebo must be at least 15% ~12 Months
(>90% powered to detect the difference)
PALISADE = Peanut ALlergy Oral Immunotherapy Study of AR101 for DEsensitization in Children and Adults
DBPCFC = Double-Blind, Placebo-Controlled Food Challenge
14 Tolerate = dose is successfully ingested with no dose-limiting symptomsAR101 Phase 3 Program:
Towards Data to Position for Market Success
PALISADE U.S.,
roll-over study to real-life setting, no oral
explore dosing intervals
ARC004 RAMSES food challenge
during maintenance N≈440
Core Approval Study
U.S., Canada, Europe
N>550 Europe,
Ages 6-48 months Broader population,
Builds on DEVIL* ARC005 ARTEMIS 1,000 mg endpoint,
study findings 3 months maintenance**
N=160
*Vickery, BP et al. Early oral immunotherapy in peanut-allergic preschool children is safe and highly effective. J Allergy Clin Immunol. January 2017
15 ** ARTEMIS is enrolling patients who tolerate ≤100 mg peanut protein at baseline; primary endpoint is tolerating a single highest dose of 1,000 mg after 3 months maintenanceAR101 Therapeutic Goal:
Personalized Up-Dosing for Dependable Protection
AR101 Treatment Protocol
Up-Dosing Phase
Ongoing Maintenance
~6 Months
Each up-dose is 300 mg
240 mg
conducted at the 300 mg Sachets
200 mg
allergist’s office 160 mg for At-Home
120 mg Daily Maintenance
80 mg Dosing
40 mg
Initial 20 mg
Escalation 12 mg
6 mg 6 mg Calendar Pack
for At-Home
3 mg
0.5 mg Dosing
• Start at a low dose (~1/500th of a peanut)
• Time between steps-ups is two weeks or longer based on individual needs
• Dosing flexibility allowed to accommodate individual and family needs
16Patients Want a Bite-Proof Level of Protection They Can Count On
• “Bite-proof” protection:
tolerate ≥ 2 peanuts
• High degree of certainty
of protection
• Rapid therapeutic effect
• Reduced symptom
severity should a reaction
to peanut exposure occur
Therapeutic goal of
AR101 aligns with
desire for confirmed
bite-proof level of
protection
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
17AR101 Dosing Would Fit Well Into Allergy Practices
~5,000 U.S. Allergists
• Allergists are highly motivated to offer approved treatments
• AR101 up-dosing is similar to allergy shot experience
• Practice capacity currently exists to integrate and scale AR101
• Expect group practices to be high adopters
~75% of U.S. allergists
surveyed by AAAAI Insights based on Aimmune sponsored market research
would prescribe an with U.S. allergists and practice managers (N=70)
FDA-approved
oral immunotherapy*
18
*Greenhawt, Vickery, JACI (2015) – Survey of members of The American Academy of Allergy, Asthma & Immunology (AAAAI)AR101 Has the Potential to Capture Meaningful Market Share
1.6 Million Potential Patients age 4-17
~1 million clinically managed today
Diagnosed
Potential referrals
and managed 30% from pediatricians Expected drivers of
by pediatricians patient motivation
to seek
AR101 AR101 therapy
AR101
Diagnosed is the only
and/or managed 70% Phase 3 • Reaction history
by allergists therapy in
development • Family support
AR101 would fit well into
for teenagers
current allergy practice • Proximity to
allergist office
70% 30%
Ages 4-11 Ages 12-17
19 Source: Symphony claims data, 2011-2016Looking Forward: Potential FDA-Approved Therapies Could
Change the Treatment Paradigm for Peanut Allergy*
2017 2025
OIT Homebrews OIT Homebrews
Clinical Trials Clinical Trials
Avoidance Only
Avoidance Only Desensitization via
Various Strategies
Potentially including
AR101 ±
adjunctive therapies,
initially for ages 4-17
20 *Proportions are illustrative and continued avoidance in all cases is expectedStrong Proprietary Position with AR101
Biologic Data Exclusivity (12 years in the U.S.)
Issued Patents Covering Formulation and Manufacturing
Exclusive Commercial Supply Agreement
Manufacturing Expertise, Trade Secrets and Know-How
First Mover Advantage and Building a Trusted Brand
21Collaborations Focused on Advancing the Field of Food Allergy
• $145M Strategic Equity Investment (Nov 2016) • Clinical Collaboration (Oct 2017) with Aimmune–
Regeneron/Sanofi Joint Development Committee
• Two-year Working Collaboration
• Phase 2 of AR101 with adjunctive dupilumab
• Aimmune Retains Full Global Rights to All expected to start in 2018*
CODIT™ Pipeline Assets, Including AR101
• Plan to explore sustained unresponsiveness in
peanut allergy
Building Pipeline Scientific Discovery
AR101 Value Expansion and Innovation
22 * Regeneron will sponsor the trial, with Aimmune to provide clinical supply of AR101 and food challenge materialsAimmune Investment Highlights
• AR101 for Peanut Allergy addresses a life-threatening disease with no approved therapies
• Robust Phase 3 program with PALISADE: final study visits expected around year-end 2017;
topline data expected 1Q 2018
• Building for 2018 regulatory filings and launch readiness in U.S. and EU
• Cutting-edge scientific collaborations including a $145 million equity investment from Nestle
Health Science and a Phase 2 clinical collaboration with Regeneron/Sanofi
• Aimmune has full global development and commercial rights for AR101 and pipeline, including
preclinical programs in egg and tree nut allergies
• Well capitalized with $212 million in cash and investments as of September 30, 2017
23Less risk. More life.
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