Safe Harbor Statement

Transcription

1 February 2012

2 Safe Harbor Statement This presentation contains certain forward-looking statements (statements as to matters other than historical facts) as defined in the Private Securities Litigation Reform Act and in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of Such statements involve risks and uncertainties that could cause actual events or results to differ materially from the events, projections or results described in the forward-looking statements. These risks and uncertainties are described in AMPE s most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K. 2

3 Ampio Background & History Over $50M contributed to product development plus ~$50M PIK May / June 2011 Listed on the NASDAQ and the Russell Index DMI Founded in 1990 by David Bar-Or, MD March 2010 DMI Merges with Chay Enterprises to Form Publicly Traded Ampio Pharmaceuticals January 2011 Commenced Clinical Trial Development on Optina September 2011 Signed First Licensing Agreement for Zertane (Korea) Sponsored research agreement with hospitals assists with substantial product development April 2011 Completed $12.7M PIPE Financing December 2011 Completed $9.4M Registered Direct Financing July 2011 Commenced Clinical Trial Development of Ampion 3

4 The Ampio Value Proposition Our pipeline consists of both novel and existing compounds strategically aimed at expected multi-billion dollar indications with limited branded competition Potentially shorter regulatory pathways that leverage the safety profile of our patent protected portfolio 3 clinical stage compounds with full rights retained in all major markets The traditional approach to drug development is broken, we believe we ve found a better path 4

5 Product Portfolio Aimed at Major Markets with Limited Existing Competition Ampion Optina Zertane Market Opportunity Innate biological anti-inflammatory product Oral therapy to prevent diabetesinduced blindness Premature Ejaculation, a larger patient population than ED Active Compound DADKP, a cyclic dipeptide derivative of human serum albumin Danazol Tramadol Clinical Stage Completed Phase II; approaching FDA in Q Human data expected Q2 2012, approaching FDA in Q Human efficacy trials completed, approaching FDA 2012 Patents Composition of matter, manufacturing, and use patents Use patents filed in the US & EU 32 use patents issued in the US & internationally, Zertane + PDE5i patents pending 5

6 Product Overview: Ampion

7 Ampion Market Potential Types of disease processes Inflammation Immune mediated Dry Eye Osteoarthritis Rhinitis Surgical wash Multiple sclerosis Crohn s Rheumatoid arthritis 25M patients in the US No good current standard of care Local application 7

8 Ampion: A Derivative of Human Serum Albumin for Osteoarthritis Patented small molecule with non-steroidal anti-inflammatory and analgesic properties that is naturally produced by humans in response to injury Discovered in trauma patients Found to be a non-steroidal biologic within human serum albumin Because it is a component of a product that has been available for over 50 years, we were able to quickly bring Ampion to human trials Human serum albumin is approved for intravenous administration and has been given to millions of patients 8

9 Ampion Anti-Inflammatory Mechanism of Action Multiple biochemical pathways identified Downmodulates T-lymphocytes pro-inflammatory cytokine production. Decreases levels of IL-23 and IL-17 (pro-inflammatory cytokines) stimulated CXCL1 production from splenic cells Decreases the response to pro-inflammatory stimuli (as demonstrated in 3T3 fibroblasts) Inhibit early activation of memory T cells but not naïve T cells Inhibits transcription factors involved in inflammation (NFkB, cjun, ATF2) Partially inhibits HMG CoA reductase (like statins anti-inflammatory effect) 9

10 Phase II: Ampion-In-Knee (AIK) Trial In Australia; patients with OA of the knee, years old, and no recent treatment, surgery or other complications 43 patient randomized, double-blinded trial, patients given a local injection of Ampion or saline Primary endpoint of pain numeric rating scale, measured at baseline, 6, 24, 72hours, 8 days, and 30 days post-injection Validated WOMAC pain & function scale measurements were also performed, data has not yet been analyzed No treatment related SAE were found in either this trial or the previous AIK trial (in 60 patients), TEAE were all minor and not different than saline 10

11 Trend Toward a Difference in Pain NRS Versus Placebo at 30 Days; Ampion Significantly Better than Baseline P=0.081 P=0.002* Analysis of covariance (ANCOVA), adjusting for baseline pain NRS Treatment group N= % improvement, 30 days P value Ampion % 0.16 Saline % *P value based upon subset of patients who did not receive rescue medication at 30 days 11

12 More Patients on Ampion Showed Improvement in Pain at 30 Days than Placebo Trends in pain at 30 days from baseline, by treatment group Change in pain Ampion Saline P value* No clinical benefit 17.7% (3) 40.0% (6) Ambivalent 17.7% (3) 20.0% (3) Improved pain 64.7% (11) 40.0% (6) Clinically meaningful improvement in pain = Decrease in 30 day pain NRS of 2 or more points Ambivalent = Decrease in 30 day pain NRS of 1 point No clinical benefit = Change in 30 day pain NRS of 0 or more points *Cochran-Armitage trend test 12

13 Ampion Regulatory Pathway Schedule meeting with FDA in Q to discuss regulatory pathway: Confirm that blood products are still considered biologics Confirm legislation for a repurposed biologic Study design for trial(s) needed for approval Begin definitive trial(s) for Ampion in osteoarthritis of the knee in 2H

14 Product Overview: Optina

15 Optina Market Potential Decrease in Inflammation Induced Vascular Permeability Diabetic Macular Edema (DME) Wet AMD Rhinitis Nephropathy 10% of all diabetics (25.8M diabetics in the US) Tablet competing against (or could be used in conjunction with) quarterly intravitreal injection 15

16 Optina: Danazol for the Treatment of Diabetic Macular Edema (DME) Optina is a very low dose of danazol, given orally, that works inside the vessels to decrease the leakage Danazol is a weak androgen that was originally approved at high doses for endometriosis, fibrocystic breast disease and hereditary angioedema (HAE) Dosing for currently approved indications is 200mg 800mg orally Use has been limited by its masculinizing side-effects At low in-vitro concentrations, danazol significantly inhibits inflammation-induced vascular permeability in human tissues Effect is reversed at higher doses 16

17 In Vitro Impact of Danazol on Vascular Permeability of Human Retinal Endothelial Cells Human Retinal Endothelial Cells Change in OD Measurement Blank 0.05 μm Danazol 0.5 μm Danazol Paradoxical Dose Effect 5.0 μm Danazol 17

18 Diabetic Macular Edema (DME) and Optina DME is a swelling of the macula in diabetic patients due to leaking of fluid from blood vessels within the macula which can cause blindness over time Prevalence is correlated with length of time with the disease 10% develop DME after 10 years, 20% after 20 years, etc Diabetes is the leading cause of new cases of blindness among adults aged years Current palliative treatment includes laser therapy and intraocular injection of Lucentis - approved for DME in EU and used off-label in US 18

19 Optina in DME Trial Design Patients with Diabetic Macular Edema (DME), 18 80yrs old, with well controlled blood sugar & no recent surgery or intra-vitreous injections Double-masked, placebo-controlled study of three orally administered doses Up to 60 patients, single site (St. Michael s Hospital, Toronto) Doses significantly less than what is currently available 3 month follow-up period Endpoints: Optical Coherence Tomography (OCT) and Visual Acuity using validated method Conducted and approved under Health Canada guidelines using selection and evaluation criteria similar to the completed Lucentis trial Data anticipated in 2Q '12 19

20 Optina Regulatory Pathway DME trial results anticipated in 2Q 2012 Mid-2012: present data to FDA to discuss pivotal trial(s) design Traditional 505(b)2 approval pathway Primary endpoint likely visual acuity, consistent with FDA guidance In parallel to US regulatory pathway, select a country to begin trials in EU Begin pivotal trial after discussions with EU regulatory body Trial design anticipated to be similar to US Primary endpoint of OCT and secondary endpoint of Visual Acuity 20

21 Optina Patents US, PCT and European patent applications for use protection have been filed Filed through PACE program in EU Accelerated review in the US No prior art teaches these uses Unanticipated activity at low doses: Inhibit vascular permeability, including that occurring in macular edema and diabetic retinopathy Modulate cytoskeleton of endothelial cells 21

22 Product Overview: Zertane

23 Zertane Market Potential Premature ejaculation (PE) is more common than erectile dysfunction (ED) and as many as 30% of men worldwide report having PE 40M patients in the US & Europe alone Global sales for ED were ~$4B in 2010 ($2B ex-us) One product has been launched to date for PE, J&J s Priligy Priligy (dapoxetine) Zertane (tramadol) Status Launched in 14 countries outside the US Phase III trials completed Pricing Prohibitively high Likely competitive with ED market pricing Marketing Janssen-Cilag, modest effort Via partners (Daewoong in Korea) Side Effect Profile Efficacy Black box warnings about suicide ideation Increased mean IELT EP over baseline by % Well established at higher doses than 62mg Increased median & mean IELT EP over baseline 237% & 407% 23

24 Zertane: Tramadol for the Treatment of Premature Ejaculation (PE) Tramadol is a centrally acting synthetic opioid analgesic that was originally approved for treating moderate to severe pain Safety record established during 30 years of human use The maximum dosage per day for currently approved indications is 400 mg for oral use and 600 mg for parenteral use, well in excess of dosage tested in clinical trials of Zertane (62 mg) Conducted successful multi-center randomized, placebo-controlled Phase III clinical trials in Europe for Zertane 62 + sites in 11 countries 604 (ITT) patients Excellent safety (no SAEs) Efficacy endpoints (all statistical objectives achieved) Intravaginal Ejaculation Latency Time (IELT) Premature Ejaculation Profile Questionnaire (PEP) 24

25 Zertane Phase III Efficacy Results Significant Increase In Median IELT P= Significant Improvements Measured By PEP Questionnaire Placebo Zertane P value vs placebo 2.75 Satisfaction with Sexual Intercourse Control over ejaculation during sexual intercourse Ejaculation-related distress < fold increase in IELT, minutes Ejaculation-related interpersonal difficulty placebo 62mg 25

26 Zertane Market Strategy Actively being pursued by country- & region-specific partners Signed first partnership deal with Daewoong of South Korea in Sept 2011 First regulatory submission to TGA in Australia, anticipated mid-2012 Will pursue U.S. approval in collaboration with a partner Funds from licensing deals may help fund company s overall development pathway 26

27 Future Directions Zertane plus PDE5 inhibitors 30% of men with ED have concomitant PE Patient population has already seen a physician and/or been treated for ED Patent on Zertane plus PDE5 inhibitors pending in 32 countries, issued in Canada (patents originally filed ) Combination product alone has become a compelling partnering opportunity Korean partner (Daewoong) committed to running a Phase III trial for a Zertane/PDE5 combination product in Korea, have started pre-ind work 27

28 Business and Financial Overview

29 Experienced Management Team David Bar-Or, MD Founder & CSO Michael Macaluso CEO Vaughan Clift, MD CRO World recognized researcher and clinician Discovered over 300 new therapeutic candidates including Optina and Ampion Head of the Trauma Research Units of two of Colorado s three Level One trauma centers Serial Entrepreneur Management experience in manufacturing, life sciences, & pharmaceuticals Experience in both the public & private sectors Lead investor DMI Ampio Pharmaceuticals Was nominated as a top scientist of the decade by Johnson Space Center Credited with creating the first FDA approved autologous cell therapy company Leading NASA scientist and physician, received numerous accolades and commendations from NASA and Lockheed Martin Deborah Knobelman, PhD CBO Strategy for Pfizer s Primary Care portfolio Wall Street Equity Research Analyst (Piper Jaffray, JPMorgan) PhD in Neuropharmacology from University of Pennsylvania Mark McGregor CFO CPA with over 30 years experience in a variety of industries Well versed in public company accounting SOX and SEC knowledgeable 29

30 Ownership and Liquidity Number of Shares Fully Diluted 35.6M Notes Shares Outstanding 31.1M Float 14.7M (2.4M in 4 Russell Indices) Mgmt Lock-up (till 7/15/12) DMI Bio (friends & family) Lock-up: 8.3M 5.9M Board 2.2M (No programmed/ 10B5 selling permitted during lock up period) Through 6/30/12 30

31 Financial Summary ($ in millions) September 30, 2011 June 30, 2011 March 31, 2011 (unaudited) (unaudited) (unaudited) Operating expenses $2.8 $1.6 $2.2 Net loss (2.6) (2.8) (8.8) Cash Debt

32 Market Exclusivity Strategy and IP Ampion Composition of matter patent that expires in 2021 Manufacturing patents (on both the filtrate and the synthetic) Multitude of use patents Optina US, PCT and European patent applications for use protection have been filed, will be filed in additional countries by end 2011 Filed through PACE program in EU No prior art teaches these uses Unanticipated activity at low doses: Inhibit vascular permeability, including that occurring in macular edema and diabetic retinopathy Modulate cytoskeleton of endothelial cells Zertane Use and formulation patents on Zertane issued in 32 countries, expire 2022 Patent on Zertane plus PDE5 inhibitors filed in 32 countries, issued in Canada Branding strategies Low dose ODT formulation 32

33 Near Term Milestones Q Q Ampion meeting with FDA Q2 Q Zertane meeting with TGA Ongoing Q Q mid Q Q Q Meet with FDA regarding Zertane regulatory pathway Optina clinical trial results in DME Meet with FDA regarding Optina pathway Ongoing Potential for additional licensing deals for Zertane to be announced 33

34 Pipeline Complete trial and 510 K submission on ORP Device Begin toxicology for methylphenidate (glioblastoma) Pursue additional indications for Optina (nephrology) Pursue additional indications for Ampion (conjunctivitis, pulmonary fibrosis, asthma, topical applications) 34 34