Corporate Presentation Fall 2015 Safe Harbor Statement Special - - PowerPoint PPT Presentation

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Corporate Presentation Fall 2015 Safe Harbor Statement Special - - PowerPoint PPT Presentation

Corporate Presentation Fall 2015 Safe Harbor Statement 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


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Corporate Presentation

Fall 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.

Safe Harbor Statement

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Investment Highlights

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

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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

Pets Humans

Development Commercial

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Regulatory Environment

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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7 AT-001 Osteoarthritis/Pain

Proof of Concept Safety & Effectiveness Chemistry, Mfg. & Control (CMC) Approval

AT-002 Inappetence AT-003 Post-Operative Pain

Proof of Concept Safety & Effectiveness Chemistry, Mfg. & Control (CMC) Approval

US US EU EU US US EU EU

2018

US US EU EU

2015 2016 2017 2014 2012 2013 Approval Chemistry, Mfg. & Control (CMC) Safety & Effectiveness Proof of Concept

Initial Product Portfolio

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Our Pipeline

Pilot Pivotal Commercial in 2016 Proof of Concept

AT-004

Monoclonal Antibody B-cell Lymphoma

AT-005

Monoclonal Antibody T-cell Lymphoma

AT-001

Osteoarthritis

AT-002

Appetite Stimulant

AT-014

Osteosarcoma

AT-003

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-008

Lymphoma

AT-018

Atopic Dermatitis

AT-002

Weight Gain

AT-002

Weight Gain

AT-007

Immunodeficiency Virus

AT-001

DJD Pain

AT-006

Feline Herpesvirus

AT-016

Allogeneic Stem Cell OA

AT-003

Post-operative Pain

AT-Iota

Periodontal

Field Studies*

* Current or next intended field study

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Our Industry

Historically, not innovation-focused

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)

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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
  • Medicines are high

margin to practice

  • Facing pressure from

alternative channels

Other Pharma 9%

Practice Revenue Mix

Commercial Environment

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Planned Sales Effort

Specialty and Primary Care

  • Direct sales force covering

approximately two dozen territories

  • Supplemented by distributors
  • Corporate sales and eCommerce
  • Alignment with veterinarians

Desire innovation

Prescribe and dispense medications

Partner mentality

  • Scalable commercial model

Unique ability when compared to human therapeutics to focus

  • n ROI given differences in payer and pharmacy environments
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Direct Veterinary Marketing

Less promotional regulations than human pharmaceuticals:

  • Rebates / Sampling
  • Hospital Merchandising
  • Advertising / Symposia

Direct to Consumer Marketing

Ability to reach pet owners with advertising and public relations:

  • Social Media
  • Cable and Print Media
  • Client Marketing Programs

Marketing Efforts

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Emerging Role of the Specialist

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-011

Parvovirus

AT-017

Lymphoma

AT-Eta AT-Iota

Periodontal

AT-Theta

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Pain Market

Multimodal 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
  • ff label
  • Acupuncture
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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 ‒ No need for routine serum hematology and chemistry monitoring

  • Technical section complete for effectiveness in dogs
  • Technical section complete for safety in dogs
  • Technical section complete for CMC in dogs
  • FDA approval in dogs and product launch anticipated in 2016
  • Pilot field study in cats underway
  • Regulatory interaction in Europe underway
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AT-001 (grapiprant)

EP4 receptor biology

Phospholipids Arachidonic Acid Cyclooxygenase (COX)-2

PGE 2 (House keeping) TXA 2 PGI 2

NSAIDs

  • GI mucosa

protection

  • Platelet

aggregation

  • Renal function

regulation EP2 EP3

Cyclooxygenase (COX)-1

EP1

PGE 2 (Inflammatory)

  • Pain and Inflammation

EP4 Receptor

Other EP Receptors

COXIBs Bs

AT-001 EP4 Receptor Antagonist

EP4 Receptor tor

  • Pain and Inflammation
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AT-001 (grapiprant)

Pivotal Dog Study Results

Design

  • Blinded, placebo-controlled, multi-site study
  • 280 client-owned dogs
  • 2 mg/kg once daily by tablet
  • Owner assessment
  • Concurred protocol with CVM

Primary Endpoint: Canine Brief Pain Inventory – Success Rate on Day 28

  • AT-001 48.1% vs. Placebo 31.3% (p<0.05)

Other Effectiveness Parameters Successes

  • Pain Severity Score (PSS) % improvement on Day 28 (p<0.01)
  • Pain Interference Score (PIS) % improvement on Day 28 (p<0.01)
  • Success rates CBPI on day 7, day 14, day 21 (p<0.05 at each)

Adverse Events

  • Only mild adverse events
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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
  • FDA approval in dogs anticipated in 2016
  • Positive pivotal field effectiveness study in dogs
  • Technical section complete for safety in dogs
  • Technical section for CMC submitted in dogs
  • Positive pilot field effectiveness study in cats enables pivotal field

effectiveness study

  • Regulatory interaction in Europe underway
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AT-003 (ER bupivacaine)

Biology

Discrete chambers within a lipid matrix, each filled with bupivacaine liposome

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AT-003 (ER bupivacaine)

Surgical technique

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AT-003 (ER bupivacaine)

Pivotal Dog Study Results

Design

  • Blinded, placebo-controlled, multi-center study
  • Approximately 150 client-owned dogs undergoing knee surgery
  • Up to 5.3 mg/kg in a single dose by infiltration
  • Veterinarian assessment
  • Concurred protocol with CVM

Primary Endpoint: Improvements in Pain Evaluation

  • AT-001 vs. Placebo (p<0.05)

Other Effectiveness Parameters Successes

  • Success (p<0.05) at each 24 hour interval up to 72 hours

Adverse Events

  • Well tolerated
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Medical Need

  • More than 20% of dogs have osteoarthritis
  • Poor compliance with giving oral drugs
  • NSAIDs cause largest amount of side effects of approved drugs
  • NSAIDs don’t work or not tolerated in significant portion of population

Our Solution

  • Stem cell therapy
  • First to market regenerative medicine product
  • Point-of-care availability as a single intra-articular injection
  • Long term relief of clinical signs (i.e pain, disability)
  • Potential regeneration of joint damage

AT-016

Allogeneic Stem Cell Production

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1.5 million Doses

  • f Final

Drug Product Vet-Stem Single Fat Donation GMP Cell Expansion

Cryostorage and Distribution Harvest , Wash, Formulate and Freeze Initial isolation and expansion in GMP process Donor fat tissue Collection

AT-016

Allogeneic Stem Cell Production

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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
  • FDA approval in dogs anticipated in mid-2016
  • Positive pivotal field effectiveness study in dogs
  • Technical section complete for safety in dogs
  • Technical section for CMC submitted in dogs
  • Positive pilot field effectiveness study in cats for weight gain enables

pivotal field effectiveness study

  • Regulatory interaction in Europe underway
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AT-002 (capromorelin)

Ghrelin biology

Brain

Pituitary gland

Stomach Intestine

Ghrelin Ghrelin

  • Increases appetite
  • Promotes growth

hormone secretion

  • Regulates energy

metabolism

  • Stimulates production of

lean body mass AT-002 Ghrelin Agonist

Growth Hormone (GH)

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AT-002 (capromorelin)

Pivotal Dog Study Results

Design

  • Double-masked, placebo-controlled, multi-site study
  • More than 200 client-owned dogs enrolled, 2:1 randomization
  • Inappetence from a variety of causes
  • 3 mg/kg once daily by liquid for 4 days
  • Concurred protocol with CVM

Primary Endpoint: Owner Appetite Assessment – Success Rate on Day 4

  • AT-002 ~70% vs. Placebo ~45% (p<0.05)

Other Effectiveness Parameters Successes

  • Alternative Owner Appetite Assessment (p<0.05)
  • Body Weight (p<0.05)

Adverse Events

  • No serious adverse events related to AT-002
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Medical Need

  • Incidence in dogs estimated at up to 10% with recent product launch (Apoquel)

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, AND stains on skin Our Solution

  • CRTH2 mechanism treats underlying disease rather than symptoms
  • Target has been validated in human medicine (asthma, allergic rhinitis and others)
  • Pilot field study underway with results expected in early 2016

AT-018

Atopic Dermatitis

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AT-018

Biology

Figures adapted from: (Left) Townley, R. G. and S. Agrawal (2012). "CRTH2 antagonists in the treatment of allergic responses involving TH2 cells, basophils, and eosinophils. "Ann Allergy Asthma Immunol 109(6): 365-374. (Right) Arima, M. and T. Fukuda (2011). "Prostaglandin D(2) and T(H)2 inflammation in the pathogenesis of bronchial asthma.“ Korean J Intern Med 26(1): 8-18.

CRTH2 Signaling AT-018 inhibits PGD2 mediated activation and recruitment of basophils, eosinophils & TH2 lymphocytes.

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Oncology Market

Highly concentrated

Board Certified Veterinary Oncologists

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Oncology

Target-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 Erbitux (EGFR) Solid Tumors Herceptin (HER2) Breast Cancer Campath (CD52) Chronic Lymphocytic Leukemia Mylotarg (CD33) Acute Myeloid Leukemia Zevalin (CD20) Follicular Lymphoma Bexxar (CD20) Non-Hodgkin's Lymphoma Vectibix (EGFR) Solid Tumors TheraCIM (EGFR) Solid Tumors

Human Chemo Market Human Cancer Antibody Market

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Pet Antibodies

A 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

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AT-004 and AT-005

Lymphoma monoclonal antibodies

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Canine Lymphoma

Attractive opportunity for first antibody therapy in dogs

  • 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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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

  • Aid in the treatment of canine B-cell lymphoma
  • USDA full license granted
  • Mini B-CHOMP underway with results expected in mid-2016
  • Three separate investigator initiated trials support use of the product in

combination with various chemotherapy regimens

  • Not as specific as desired to fully capture the market

AT-004

Canine-specific antibody for B-cell lymphoma

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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
  • USDA conditional license received
  • T-CHOMP and T-LAB not expected to show survival benefit with final

results expected in mid-2016

  • Not as specific as desired to fully capture the market but currently the
  • nly available monoclonal antibody

AT-005

Canine-specific antibody for T-cell lymphoma

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AT-014

Therapeutic vaccine for canine 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
  • USDA conditional license anticipated in 2016
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AT-014

Biology

  • 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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AT-014

Preliminary Efficacy Data

All dogs without gross metastatic disease at the time of first dosing

Median survival Control: 316 days Canine OSA Vaccine: 956 days

Diagnosis & Amputation Chemotherapy (Carboplatin 300mg/m2 q 21 days x 4) Screening visit 3 vaccinations given 3 weeks apart Re-staging visit 3 weeks after last vaccination Re-staging visits q2months

Boosters provided

  • ngoing as needed

every 4- 6 months n=18 p < 0.0001

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Other Pipeline Products

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-017 Dog Lymphoma Lead selection Other: AT-epsilon, AT-zeta, AT-iota, AT-eta, AT-theta

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Upcoming Milestones

  • Technical section complete for dog efficacy (AT-002, AT-003)
  • Technical section complete for dog CMC (AT-002, AT-003)
  • NADA submission for dogs (AT-001, AT-002, AT-003)
  • Triggers 60 day timeline to anticipated approval
  • Commercial launches in 2016
  • Results of pilot studies (AT-001 Cat 4Q2015, AT-018 Dog 1Q2016)
  • Initiation of pivotal studies (AT-016 Dog, AT-002 Cat, AT-003 Cat)
  • OUS partnering
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Our Financial Profile

  • Cash balance $83.6M at June 30, 2015
  • Net Loss quarter ending June 30, 2015 was ($8.0M) or ($0.23)/shr
  • Anticipate being at low end of 2015 guidance of $45M- $55M cash

used in operations

  • Cash and equivalents sufficient to fund operations through 2016
  • Continue to identify non-dilutive sources of capital

‒ Debt instruments ‒ Partnering opportunities globally ‒ Monetize non-core products and operations

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Investment Highlights

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