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Special Note Regarding Forward-Looking StatementsThis presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements with respect to expectations regarding the timing of discussions with the FDA and/or USDA and approval and licensure of products; development programs, clinical trials and studies, including without limitation the timing of full enrollment in and the announcement of results of such trials and studies; commercialization and manufacturing of products, including without limitation establishing larger manufacturing capacities for AT-004 and AT-005; the sufficiency of financial resources; expected future cash balance and liquidity; licensing initiatives and collaborations; the Company’s plans and opportunities, including without limitation offering a unique portfolio of innovative therapeutics; and the Company’s belief that its products and product candidates will result in improved outcomes for pets.
These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our limited operating history and expectations of losses for the foreseeable future; our lack of commercial sales; our failure to obtain any necessary additional financing; market conditions and our ability to raise capital under the shelf registration statement from the sale of our securities; our substantial dependence on the success of certain of our lead product candidates; our dependence on novel technologies and compliance with complex regulatory requirements; our inability to obtain regulatory approval for our existing or future product candidates; the lack of commercial success of our current or future product candidates; our inability to realize all of the anticipated benefits of our acquisitions of Vet Therapeutics and Okapi Sciences; uncertainties regarding the outcomes of studies regarding our products; the uncertainty of outcomes of the development of pet therapeutics, which is a lengthy and expensive process; effects of competition; our inability to identify, license, develop and commercialize additional product candidates; our failure to attract and keep senior management and key scientific personnel; our reliance on third-party manufacturers, suppliers, partners and other third parties which conduct our target animal studies and certain other development efforts; unanticipated difficulties or challenges in the relatively new field of biologics development and manufacturing; our ability to market our products only for the treatment of indications for which they are approved; our small commercial organization; difficulties managing the growth of our organization; our significant costs of operating as a public company; risks related to the restatement of our financial statements for the year ended December 31, 2013 and the identification of a material weakness in our internal control over financial reporting; changes in distribution channels for pet therapeutics; consolidation of our customers; limitations on our ability to use our net operating carryforwards; impact of generic products; unanticipated safety or efficacy concerns; our limited patents and patent rights; our failure to comply with our intellectual property license obligations; our infringement of third party patents and challenges to our patents or rights; litigation resulting from the misuse of our confidential information; the uncertainty of the regulatory approval process; our failure to comply with regulatory requirements or obtain foreign regulatory approvals; our failure to report adverse medical events related to our products; legislative or regulatory changes; the volatility of our stock price; our status as an “emerging growth company,” as defined in the JOBS Act; the potential for dilution if we sell shares of our common stock in future financings; the significant control over our business by our principal stockholders and management; the potential that a significant portion of our total outstanding shares could be sold into the market in the near future; effects of anti-takeover provisions in our charter documents and under Delaware law; and our intention not to pay dividends. These and other important factors discussed under the caption "Risk Factors" in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission, or SEC, on March 16, 2015, along with our other reports filed with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this presentation. Any such forward-looking statements represent management's estimates as of the date of this presentation. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this presentation.
Safe Harbor Statement
3
Investment Highlights
Large, growing market
De-risked drug development
Private-pay
Portfolio approach
Scalable and capital-efficient
First mover, pure-play
4
Defining Pet Therapeutics
2010 2013 2014
Founded
Completed IPO 3 products in development
Portfolio Expansion: Option Deals Vet Therapeutics Okapi Sciences Advaxis Vet-Stem Atopix
Over 18 products in development
Introduction of Lymphoma MAb
2015 2016
Regain Rights to AT-004
5
Source: APPA.
Our Market
Large and growing
– 2014 U.S. pet spend of $58B
Insensitive to recent recessions
68% of U.S. households
– 96M cats
– 83M dogs
Medicalization drives growth
– Historically, limited innovation
– Pet therapeutics underrepresented
U.S. Pet Owners Spend($Billions)
6
The Evolution of Pet Ownership
“Snoopy Generation” “Brian Generation”
Pets are family whose medical needs merit quality care
7
A Favorable Comparison
Multiple Species Steps Direct to Species
~$1.3B ~$10M
~10 Years ~5 Years
Third Party Payer Private Pay
Generic Pressure Innovator Brand Loyalty
Difficult & Indirect Accessible & Direct
PetsHumans
De
velo
pm
ent
Co
mm
erc
ial
8
Building the Portfolio
Toxicology data are available
Manufacturing is scaled-up
Effectiveness data are available
Key Attributes
Unmet medical need
High incidence or prevalence
Known mechanism of action
Nuance in science
“Early de-risking”
Early De-Risking
Leveraging the investment in human therapeutics Pharmaceuticals Biologics US and OUS
9
FDA and USDA TimelinesFDA Center for Veterinary Medicine
Proof of Concept
INAD
Chemistry, Mfg. & Controls (CMC)
Safety
Effectiveness
Labeling, FOI Summary, Other
Administrative NADA
Year 5Year 1 Year 2 Year 3 Year 4
USDA - Center for Veterinary Biologics
Proof of Concept
Manufacturing
File for Product License
Preclinical
Field Safety and Efficacy
Conditional Product License*
Extended Field Safety and Efficacy Study
Full Product License* Conditional l i censes granted under specia l ci rcumstances
Year 5Year 1 Year 2 Year 3 Year 4
Pilot ---> Pivotal
10
Our Pipeline
PilotPivotal/
CommercialProof ofConcept
AT-004Monoclonal Antibody
B-cell Lymphoma
AT-005Monoclonal Antibody
T-cell Lymphoma
AT-001(grapiprant)
Osteoarthritis
AT-002(capromorelin)
Appetite Stimulant
AT-014Osteosarcoma
AT-003(bupivacaine liposome injectable suspension)
Post-operative Pain
AT-012Feline Calicivirus
AT-009Mast Cell Tumor
AT-011Canine Parvovirus
AT-010Atopic Dermatitis
AT-015Lymphoma
AT-017Lymphoma
AT-BetaEpilepsy
AT-008Lymphoma
AT-018Atopic Dermatitis
AT-002(capromorelin)
Weight Gain
AT-002(capromorelin)
Weight Gain
AT-007Feline Immunodeficiency Virus
AT-001(grapiprant)
Degenerative Joint Disease
AT-006Feline Herpesvirus
AT-016Allogeneic Stem Cell OA
AT-003(bupivacaine liposome injectable suspension)
Post-operative Pain
AT-IotaPeriodontal
11
Our Industry
Total number of NADAs NADAs for dogs / cats Pet therapeutic NCEs*
2011 12 6 2
2012 11 6 0
2013 6 4 2
2014 11 4 0
* NCE defined as new chemical entity not previously approved in humans or animals (excluding parasite drugs).
Non-confidential materials
(may not be comprehensive)
~15 ~3
~1
~2
~1
Products at Field Study Stage in Dogs and Cats
Historic Productivity of the Animal Health Industry
13
Annual Exams17%
Diagnostics17%
Surgery17%Non-Invasive
Procedures9%
Pet Food Sales4%
Heartworm Product Sales
6%
Flea-Tick Product Sales
6%
Vaccinations15%
Source: DVM Newsmagazine’s State of the Profession Report 2012.
Innovation is valued and desired
Medicines are high margin to practice
Facing pressure from alternative channels
Other Pharma 9%
Practice Revenue Mix
Commercial EnvironmentAlignment with the Veterinarian
14
Therapeutic Clusters
Oncology/Specialty Pain/Primary Care
AT-016Allogeneic Stem Cell
AT-003Post-operative Pain
AT-007Immunodeficiency Virus
AT-001Osteoarthritis
AT-006Herpesvirus
AT-004B-cell Lymphoma
AT-005T-cell Lymphoma
AT-002Appetite Stimulant
AT-014Osteosarcoma
AT-018Atopic Dermatitis
AT-012Feline Calicivirus
AT-009Mast Cell Tumor
AT-010Atopic Dermatitis
AT-EpsilonMelanoma
AT-ZetaHemangiosarcoma
AT-015Cat Lymphoma
AT-BetaEpilepsy
AT-011Parvo Virus
AT-017Lymphoma
AT-EtaAnti HER-2 MAb
AT-IotaPeriodontal
16
OncologyTarget-rich, known biology
Chemotherapy Human Cancer Use Pet Cancer Use
Cyclophosphamide Lymphomas MM, solid tumors
Doxorubicin Lymphomas MM, solid tumors
Vincristine Lymphomas MM, solid tumors
Prednisone Lymphomas MM, solid tumors
L-asparaginase Leukemia, Lymphoma
Carboplatin Solid Tumors
Cis-platinum Sarcoma, Carcinoma, Lymphoma
Mitoxantrone Breast cancer, AML, Lymphomas
Lomustine Brain/CNS, Lymphoma, Mast cell
Methotrexate Lymphomas Osteosarcoma
Antibody Human Cancer Use Pet Cancer Use
Rituxan (CD20) Non-Hodgkin's Lymphoma
Avastin (VEGF) Solid Tumors X
Erbitux (EGFR) Solid Tumors X
Herceptin (HER2) Breast Cancer X
Campath (CD52) Chronic Lymphocytic Leukemia
Mylotarg (CD33) Acute Myeloid Leukemia X
Zevalin (CD20) Follicular Lymphoma X
Bexxar (CD20) Non-Hodgkin's Lymphoma X
Vectibix (EGFR) Solid Tumors X
TheraCIM (EGFR) Solid Tumors X
Human Chemo Market
Human Cancer Antibody Market
17
Pet AntibodiesA proprietary platform
Pet specific antibodies
Pet Fc region
Most effective IgG sequence
Straightforward engineering with no shuffling
IP position directed at platform
Mouse Pet Specific
Highly specific‒ Developed against pet targets
Non immunogenic‒ Compatible with immune system
Highly potent‒ Engages immune system
Cost effective‒ High yield production
heavy chain
light chain
VH
VL CH1
CH2
CH3
CL
Proprietary Platform Pet Specific Antibody Ideal Profile
18
Oncology MarketSpecialty approach
4Q14 1H15 2H15 2H161H16
Veterinary Cancer Society
T-Cell MAb Full License
T-CHOMP and T-LAB Studies
T-CEP Experience Program
~ Three dozen Participating
Practices
~75 Participating
Practices
Additional Oncology Practices
B-Cell Studies
Veterinary Cancer Society
T-CHOMP/T-LAB Results
Scientific Studies
19
Pain MarketMultimodal management
Disease Progression
Osteoarthritis
Over the counter Coxib NSAIDs EP4 MAbs, Capsaicin
Regenerative /Disease modifying
Pre / Post Surgical
Anesthesia Fentanyl Bupivacaine Bupenorphrine
Cancer
Opioids Intrathecal
Neuropathic and Other
Human drugs used off label
Acupuncture
20
American College of Veterinary Internal MedicineJune 3-6 Schedule of Events
The Evolving Science of Immunotherapeutics in Veterinary Oncology A Prospective, Open-Label Study Evaluating Treatment of Canine B-cell Lymphoma with L-Asparaginase,
Doxorubicin, and a Canine Anti-CD20 Monoclonal Antibody Clinical Application of Immunotherapeutics in Veterinary Oncology Anti-CD20 Monoclonal Antibody Canine Lymphoma Therapy: A Double-Blind, Randomized, Placebo-
Controlled Study Combination Listeria-Based Immunotherapy Plus Palliative Radiation Delays Tumor Progression and
Prolongs Overall Survival in Canine Osteosarcoma
Cancer therapies
Safety of the Ghrelin Agonist, Capromorelin, Administered Daily to Beagle Dogs for One Year Capromorelin, An Orally Active Ghrelin Agonist Caused Sustained Increases in IGF-1, Increased Food Intake
and Body Weight in Cats Safety of the Ghrelin Agonist, Capromorelin Administered Daily to Cats for 91 Days at an Oral Dose of
6mg/kg New Therapeutic Advances in Inappetence for Pets
Inappetence
Introduction to the Emerging Piprant Therapeutic Class of Prostanoid Receptor Antagonists Safety and Toxicokinetic Profiles in Cats Administered Grapiprant, A Selective Prostaglandin-Receptor
Antagonist
Pain Management
21
Anticipated Upcoming Events
2015 ACVIM Forum (multiple abstracts and scientific presentations)
Results of AT-002 Dog US pivotal field effectiveness study
Results of AT-003 Dog US pivotal field effectiveness study
Results of pilot studies (AT-016 Dog, AT-003 Cat, AT-002 Cat, AT-003 Cat)
Initiation of pilot studies (AT-017, AT-018)
Continued AT-004/AT-005 availability
Full USDA license for AT-005
22
Our Financial Profile
March 31, 2015 cash balance $87.4M
Net Loss for 1st Quarter ending March 31, 2015 was ($8.8M) or ($0.26)/ share
R&D investment will increase as a result of advancing pipeline
Continue to identify non-dilutive sources of capital‒ Partnering opportunities globally
‒ Out-licensing of non-core products
‒ Debt instruments
Strong and supportive investor base
23
Investment Highlights
Portfolio approach
Scalable and capital-efficient
First mover, pure-play
Large, growing market
De-risked drug development
Private-pay
25
AT-001 (grapiprant)For osteoarthritis pain
Medical Need
Established market (U.S. sales $260M, mostly NSAIDs for dogs)
Existing NSAID products have side effects and require monitoring
Better tolerated product for pain and inflammation of osteoarthritis
Our Solution
EP4 Prostaglandin Receptor Antagonist
‒ Potential for significantly improved tolerability profile vs. Coxibs
Positive results from pivotal field effectiveness study in December 2014
Technical section complete for safety received; CMC submitted
FDA approval anticipated in 2016
27
AT-002 (capromorelin)For inappetence
Medical Need
No currently approved product
Effective appetite stimulus to avoid feeding tubes and euthanasia
Seen in aging and chronic conditions
Our Solution
Mimics ghrelin (hunger hormone) to turn on appetite
Statistically significant results on increased appetite and weight gain in dog pilot study
Pivotal field study results expected in late-June 2015
Technical section complete for safety received; CMC submitted
FDA approval anticipated in 2016
29
AT-003 (ER bupivacaine)For post-operative pain
Medical Need
Pain increasingly recognized and treated
Need for long-acting, non-narcotic post-operative pain relief
Our Solution
Bupivacaine liposome injectable suspension
Pacira launched product for human use in early 2012
Announced positive results from pilot field study in 3Q 2014
Pivotal field study results expected in July 2015
Technical section complete for safety received; CMC submitted
FDA approval anticipated in 2016
31
Canine LymphomaIdeal for first antibody therapy
A canine-specific antibody therapy could achieve in dogs what Rituxan has done for human lymphoma
Lymphoma is most common blood cancer in dogs
- 8% of total dog cancer
Lymphoma is the most treated cancer
- Chemotherapy is the current “standard of care”
- Treatment cost ranges between $2,500 and $10,000
Clinical manifestations are similar to humans
Lymphocyte targeting has proven efficacious in pets
HemangiosarcomaSarcoma
Lymphoma
Mast Cell
Skin
BoneOther
RITUXAN-LIKE APPROACH
Incidence
33
Medical Need
76% of all lymphoma is B-cell lymphoma
Chemo achieves short remissions and has a very high relapse rate
Chemo can be harsh on pets and burdensome on owners
Need effective and safe therapy to maintain remission as long as possible
Our Solution
First-in-class product
Aid in the treatment of canine B-cell lymphoma
USDA full license granted
Planning additional scientific studies and clinical studies under field conditions in combination with chemotherapy
Second generation product with increased specificity and ability to access the OUS markets
AT-004Canine-specific antibody for B-cell lymphoma
35
A-004 for B-Cell LymphomaProgression Free Survival for Phase 1 - Updated on 26Mar14
Surv
ival D
istr
ibution F
unction
0.00
0.25
0.50
0.75
1.00
pfs1
0 100 200 300 400 500 600
STRATA: treat=Mab Censored treat=Mab treat=Placebo
------ : Placebo arm: MAb arm
Days
Pro
gres
sio
n F
ree
Surv
ival
Dogs treated with anti-CD20 MAb + CHOP (1 cycle) had statistically significant longer median PFS times than those treated with CHOP (1 cycle) alone
G.K. Ogilvie, D.R. Proulx, L. VanHorn, et al. Treatment of Canine B-Cell Lymphoma with Chemotherapy and a Canine Anti-CD20 Monoclonal Antibody: A Prospective Double-Blind, Randomized, Placebo-Controlled Study. Abstract presented at VCS 2014 Conference, St. Louis, MO.
p<0.0001
36
Medical Need
24% of all lymphoma is T-cell lymphoma
T-cell lymphoma is more aggressive and less responsive to treatment
Our Solution
First-in-class product
Aid in the treatment of canine T-cell lymphoma
Submitted for USDA product license; conditional license received in January 2014
Initiated T-CHOMP, T-LAB and T-CEP studies under field conditions in combination with chemotherapy
Additional scientific studies
AT-005Canine-specific antibody for T-cell lymphoma
37
AT-014Therapeutic vaccine for osteosarcoma
Medical Need
Estimated 8,000 to 20,000 dogs in US are affected annually
Standard of care is amputation and post-operative chemotherapy
Nine-to-twelve months median survival; only 25% of dogs survive two years
Need for effective and safe therapy with longer survival time
Our Solution
First-in-class product to aid in the treatment of osteosarcoma
Developed at University of Pennsylvania by Advaxis
Statistically significant survival post amputation
Filed for USDA approval conditional license
38
AT-014Biology
Live Vector Accesses Antigen Presenting Cells
TAA-Fusion Peptide Secreted
Triggers Innate and Adaptive Pathogen Immune Response
Tumors Now “Seen” As Pathogen-Infected and Targeted By T-Cells
Lm-LLO Immunotherapy Infusion
MHC
II
MHC
I
CD4+ T
Cell
CD8+ T
Cell
LLO mediated
escape
Activated
Dendritic Cell
tLLO-TAA Fusion
Proteins
39
Medical Need
Incidence in dogs estimated at up to 10% with recent product launch peak sales estimated at $200M
Chronic condition which often can onset at a young age (1-3 years old)
Owners can easily diagnose symptoms including itching, sneezing, hair loss, paw licking, stains on skin etc.
Our Solution
CRTH2 mechanism treats underlying disease rather than symptoms
Target has been validated in human medicine (asthma, allergic rhinitis and others)
AT-018Atopic Dermatitis
41
De-risking StrategiesDose-finding and pilot studies pivotal study
AT-001 (Grapiprant) An oral, once-daily EP4 receptor antagonist
Pilot field study completed in 2013
Clinical success rates based on the validated CBPI at day 28 were 48.1% for the once-daily dose of AT-001 vs. 31.3% for the placebo group which represents a statistically significant difference (p<0.05)
The Pain Severity Score and the Pain Interference Score both improved significantly on day 28 (p<0.01)
The clinical success rates on days 7, 14 and 21 vs. placebo group were all statistically significant (p<0.05)
In January 2015, Aratana received the target animal safety technical section complete letter for AT-001 in dogs from the CVM
Anticipate approval in 2016
42
AT-003 (ER Bupivacaine) Post-operative pain Multi-site, placebo-controlled study
in 46 dogs in an orthopedic indication
Better pain control for up to 72 hours based on pain score and time-to-rescue (p<0.05)
In January 2015, Aratana received the target animal safety technical section complete letter for AT-003 in dogs from the CVM
Dog pivotal field effectiveness study complete (protocol concurrence)
Top line results 2H July 2015
De-risking StrategiesDose-finding and pilot studies pivotal study
AT-002 (Capromorelin) Ghrelin agonist for appetite and
weight gain
Multi-site, pilot field study in dogs (17 treated, 12 placebo)
Appetite score on Day 6: 79 vs. 22 placebo (p=0.025); Body weight 3.2% vs. -0.5% placebo (p=0.024)
Dog pivotal field effectiveness study complete (protocol concurrence)
Top line results late June 2015
43
AT-005 (T-cell Lymphoma) Monoclonal antibody to aid in the
treatment of canine T-cell lymphoma
Medical science liaisons rather than general sales strategy
Targeting specialist oncologists
T-CHOMP, T-LAB and T-CEP experience studies to better understand the market and how the product will be used
T-Chomp enrollment completed
Anticipating full license from the USDA in 2015
AT-014 (Cancer Vaccine) Canine osteosarcoma
Study in 18 client-owned dogs
MST not yet reached for treated group; 80% of dogs surpassing the MST of the control group (p<0.001) and 60% still alive
USDA accepted the efficacy data to support conditional licensure
Transferring manufacturing to USDA-licensed manufacturer
Anticipate receipt of conditional USDA license in 2016
De-risking StrategiesRegulatory and post-marketing studies
44
Other Pipeline ProductsProduct Species Indication Development Status
AT-006 Cat Herpesvirus Designing pivotal study with partner
AT-007 Cat Immunodeficiency virus Pilot studies
AT-008 Dog Lymphoma Planning for pivotal studies in Europe
AT-009 Dog Mast cell tumor Lead selection
AT-010 Dog Atopic dermatitis Lead selection
AT-011 Dog Parvovirus Lead selection
AT-012 Cat Calicivirus Lead selection
AT-015 Cat Lymphoma Proof of concept
AT-016 Dog Osteoarthritis Pilot studies
AT-017 Dog Lymphoma Lead selection