Corporate Presentation 2Q 2015 Sa fe Ha rbor Sta te me nt Special - - PowerPoint PPT Presentation

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Corporate Presentation 2Q 2015 Sa fe Ha rbor Sta te me nt Special Note Regarding Forward Looking Statements This presentation contains forward looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.


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Corporate Presentation

2Q 2015

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Special Note Regarding Forward‐Looking Statements This 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

  • f 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;

  • ur 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

  • bligations; 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

  • ther 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.

Sa fe Ha rbor Sta te me nt

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Inve stme nt Hig hlig hts

  • Large, growing market
  • De‐risked drug development
  • Private‐pay
  • Portfolio approach
  • Scalable and capital‐efficient
  • First mover, pure‐play
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De fining Pe t T he ra pe utic s

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

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Source: APPA.

Our Ma rke t

  • 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)

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T he E volution of Pe t Owne rship

“Snoopy Generation” “Brian Generation”

Pets are family whose medical needs merit quality care

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A F a vora ble Compa rison

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

Pets Pets Humans Humans

Development Development Commercial Commercial

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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
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F DA a nd USDA T ime line s

FDA Center for Veterinary Medicine

Proof of Concept INAD Chemistry, Mfg. & Controls (CMC) Safety Effectiveness Labeling, FOI Summary, Other Administrative NADA Year 5 Year 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 licenses granted under special circumstances

Year 5 Year 1 Year 2 Year 3 Year 4

Pilot ‐‐‐> Pivotal

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Our Pipe line

Pilot Pivotal/ Commercial Proof of Concept

AT‐004

Anti‐CD20 Monoclonal Antibody B‐cell Lymphoma

AT‐005

Anti‐CD52 Monoclonal Antibody T‐cell Lymphoma

AT‐001

(grapiprant) Osteoarthritis

AT‐002

(capromorelin) Appetite Stimulant

AT‐014

Osteosarcoma

AT‐003

(bupivacaine liposome injectable suspension) Post‐operative Pain

AT‐012

Feline Calicivirus

AT‐009

Mast Cell Tumor

AT‐011

Canine Parvovirus

AT‐010

Atopic Dermatitis

AT‐015

Lymphoma

AT‐017

Lymphoma

AT‐Beta

Epilepsy

AT‐008

Lymphoma

AT‐018

Atopic Dermatitis

AT‐002

(capromorelin) Weight Gain

AT‐002

(capromorelin) Weight Gain

AT‐007

Feline Immunodeficiency Virus

AT‐001

(grapiprant) Degenerative Joint Disease

AT‐006

Feline Herpesvirus

AT‐016

Allogeneic Stem Cell OA

AT‐003

(bupivacaine liposome injectable suspension) Post‐operative Pain

AT‐Iota

Periodontal

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Our Industry

Total number of NADAs NADAs for dogs / cats Pet therapeutic NCEs* 2011 12 6 2 2012 11 6 2013 6 4 2 2014 11 4

* 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

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Industry Re c og nition

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Annual Exams 17% Diagnostics 17% Surgery 17% Non‐Invasive Procedures 9% Pet Food Sales 4% Heartworm Product Sales 6% Flea‐Tick Product Sales 6% Vaccinations 15%

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

Comme rc ia l E nvironme nt

Alig nme nt with the Ve te rina ria n

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T he ra pe utic Cluste rs

Oncology/Specialty Pain/Primary Care

AT‐016

Allogeneic Stem Cell

AT‐003

Post‐operative Pain

AT‐007

Immunodeficiency Virus

AT‐001

Osteoarthritis

AT‐006

Herpesvirus

AT‐004

B‐cell Lymphoma

AT‐005

T‐cell Lymphoma

AT‐002

Appetite Stimulant

AT‐014

Osteosarcoma

AT‐018

Atopic Dermatitis

AT‐012

Feline Calicivirus

AT‐009

Mast Cell Tumor

AT‐010

Atopic Dermatitis

AT‐Epsilon

Melanoma

AT‐Zeta

Hemangiosarcoma

AT‐015

Cat Lymphoma

AT‐Beta

Epilepsy

AT‐011

Parvo Virus

AT‐017

Lymphoma

AT‐Eta

Anti HER‐2 MAb

AT‐Iota

Periodontal

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Onc olog ists

Initia l c ustome r

Board Certified Veterinary Oncologists

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Onc olog y

T a rg e t- ric h, known biolog y

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 AT‐004 Avastin (VEGF) Solid Tumors X Erbitux (EGFR) Solid Tumors X Herceptin (HER2) Breast Cancer X Campath (CD52) Chronic Lymphocytic Leukemia AT‐005 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

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Pe t Antibodie s

A proprie ta ry pla tform

  • 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 pet immune system

  • Highly potent

‒ Engages pet immune system

  • Cost effective

‒ High yield production

heavy chain light chain VH VL CH1 CH2 CH3 CL

Proprietary Platform Pet Specific Antibodies

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Onc olog y Ma rke t

Spe c ia lty a pproa c h

4Q14 1Q15 2Q15 3Q15 4Q15 2Q16 1Q16 Veterinary Cancer Society T‐Cell MAb Full License T‐CHOMP and T‐LAB Studies T‐CEP Experience Program ~ Three dozen Participating Practices ~50 Participating Practices ~75 Participating Practices Open to all 200 Oncology Practices B‐Cell Studies Veterinary Cancer Society T‐CHOMP/ T‐LAB Results

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Pa in Ma rke t

Multimoda l ma na g e me nt

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
  • ff label
  • Acupuncture
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Ame ric a n Colle g e of Ve te rina ry Inte rna l Me dic ine

June 3- 6 Sc he dule of E ve nts

  • 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

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Antic ipa te d Upc oming E ve nts

  • 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 expansion of AT‐004/AT‐005 availability
  • Full USDA license for AT‐005
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Our F ina nc ia l 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
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Inve stme nt Hig hlig hts

  • Portfolio approach
  • Scalable and capital‐efficient
  • First mover, pure‐play
  • Large, growing market
  • De‐risked drug development
  • Private‐pay
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Product Detail

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AT

  • 001 (g ra pipra nt)

F

  • r oste oa rthritis pa in

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
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AT

  • 001 (g ra pipra nt)

E P4 re c e ptor biolog y

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AT

  • 002 (c a promore lin)

F

  • r ina ppe te nc e

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
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AT

  • 002 (c a promore lin)

Ghre lin biolog y

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AT

  • 003 (E

R bupiva c a ine )

F

  • r post- ope ra tive pa in

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
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AT

  • 003 (E

R bupiva c a ine )

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Ca nine L ymphoma

Ide a l for first a ntibody the ra py

  • 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

Hemangiosarcoma Sarcoma Lymphoma Mast Cell Skin Bone Other

RITUXAN‐LIKE APPROACH

I n c i d e n c e

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AT

  • 004 a nd AT
  • 005

L ymphoma monoc lona l a ntibodie s

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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 studies under field conditions in combination with

chemotherapy

AT

  • 004

Ca nine - spe c ific a ntibody for B- c e ll lymphoma

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Huma n E

xpe rie nc e

MAbs a re now sta nda rd of c a re

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A- 004 for B- Ce ll L ymphoma

S urv iv al D is tribution Func tion

0.00 0.25 0.50 0.75 1.00 100 200 300 400 500 600

‐‐‐‐‐‐ : Placebo arm : MAb arm

Days Progression Free Survival 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

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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

AT

  • 005

Ca nine - spe c ific a ntibody for T

  • c e ll lymphoma
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AT

  • 014

T he ra pe utic va c c ine for oste osa rc oma

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
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AT

  • 014

Biolog y

  • 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

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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

  • 018

Atopic De rma titis

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AT

  • 018

Role of T h2 Ce llula r Immunity

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De - risking Stra te g ie s

Dose - finding a nd pilot studie s  pivota l 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
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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 now underway (protocol concurrence)

De - risking Stra te g ie s

Dose - finding a nd pilot studie s  pivota l 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 now underway (protocol concurrence)

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AT‐005 (CD‐52 MAb)

  • 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 Stra te g ie s

Re g ula tory a nd post- ma rke ting studie s

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Othe r Pipe line Produc ts

Product 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