Ampio Pharmaceuticals, Inc. · OTC:AMPE
Ampio Pharmaceuticals — Investor Presentation (January 2018)
28 slides from Ampio Pharmaceuticals's investor presentation, Jan 8, 2018
Slide 1: Ampion Management Presentation January 2018
Ampion TM Management Presentation January 2018
Slide 2: Forward Looking Statement
Forward Looking Statement These slides and materials, including any accompanying oral presentation, contain forward-looking statements about our business. You should not place undue reliance on forward-looking statements as these statements are based upon our current expectations, forecasts and assu
Slide 3: Executive Summary
• Ampion™ is a novel biologic set to address an unmet medical need and significant treatment gap in severe osteoarthritis of the knee (OAK) ─ FDA acknowledged there are no licensed or approved therapies to address this population • Ampion has successfully completed two pivotal Phase 3 trials for the
Slide 4: Severe OAK is a serious, debilitating condition with a high unmet need
Severe OAK is a serious, debilitating condition with a high unmet need Osteoarthritis Epidemiology Report. DataMonitor, Inc. 2013. 4 Relationship between patient - reported disease severity in osteoart hritis and self - reported pain, function and work productivity. Sadosky et al. Arthritis Research
Slide 5: A high need exists to more effectively manage patients with no treatment options
A high need exists to more effectively manage patients, who have no treatment options with demonstrated efficacy, prior to, or prolonging, progression requiring TKA Crichton B, Green M. GP and patient perspectives on treatment with non - steroidal anti - inflammatory drugs for the treatment of pa in
Slide 6: IA injections limited by poor efficacy and long-term safety
IA injections are a mainstay of managing moderate OAK but are limited by poor efficacy and long - term safety and have not been evaluated for severe (KL4) patients Ampion is the only therapy to demonstrate both pain and function efficacy in severe OAK patients Common Treatment Options Used for Sever
Slide 7: Corticosteroids and hyaluronic acid evidence
Corticosteroids Hyaluronic Acid • May 2017 study from Tufts Medical Center and Boston University School of Public Health • Intra - articular triamcinolone led to significantly higher cartilage volume loss compared with saline – Mean change in index compartment cartilage thickness ( - 0.21mm vs - 0.1
Slide 8: Patients ultimately require total knee arthroplasty
Projections of primary and revision hip and knee arthroplasty in the United States from 2005 to 2030, 2007 / Revision rates a fte r total joint replacement: cumulative results from worldwide joint register datasets 2011 / UptoDate / Revision rates after total joint replacement Journal of Bone & Join
Slide 9: Economic Burden of Total Knee Arthroplasty
Total cost of hospital expenditures due to TKA* $35 Billion Failure to adequately treat severe OAK patients has resulted in a large and growing economic burden due to TKA Total charges per TKA patient (hospitalization, implant, fee) $49,500 Total charges per TKA revision surgery (12% within 10 years
Slide 10: Physicians identify a significant need in severe OAK
Physicians identify a significant need in severe OAK – Ampion is well positioned to address this treatment gap Need for improved efficacy in moderate/severe patients Pain reduction : • Conflicting evidence to support pain reduction benefit of IA hyaluronic acid and corticosteroids • Diminishing pain
Slide 11: AMPION: first-in-class injectable biologic for severe OAK
AMPION TM A first - in - class, injectable biologic for the treatment of the signs and symptoms of severe OAK A low molecular - weight filtrate of FDA - approved Human Serum Albumin (HSA)
Slide 12: Ampion has successfully completed two pivotal studies
Ampion is a first - in - class, injectable biologic treatment for the signs and symptoms of severe OA of the knee and has successfully completed two pivotal studies • Ampion is a low molecular weight (<5 kDa) ultra filtrate of 5% commercial human serum albumin for the treatment of signs and symptoms
Slide 13: Pivotal and supportive study design
Ampion has completed two pivotal studies required for FDA filing as well as several additional studies supporting efficacy as both a single and repeat injection Study Design Pivotal Studies AP - 003 - A Phase 3, n=329 (Mar 2013 - Aug 2013) • Endpoint : WOMAC A pain reduction at 12 weeks compared to
Slide 14: AP-003-A Primary Endpoint: reduction of pain by severity
Primary Endpoint In its first statistically significant pivotal study, AP - 003 - A, Ampion demonstrated reduction of pain across all OAK severities 44% 44% 35% 42% 40% 28% 18% 29% Mild/Mod (KL 2) Moderate (KL 3) Severe (KL 4) Combined FDA has provided confirmation that AP - 003 - A can be used as o
Slide 15: AP-003-C OARSI response endpoint supports signs and symptoms label
AP - 003 - C’s OARSI response primary endpoint differentiates Ampion’s label to include ‘signs and symptoms’ as a result of demonstrated effect on all key OAK endpoints • Existing therapies approved for OAK are labeled for the “treatment of pain” due to inability to demonstrate improvements in funct
Slide 16: Ampion Efficacy for Primary and Secondary Endpoints of AP-003-C
Primary Endpoint OARSI Response OARSI Controlled Responder PGA Responder Controlled Responder vs. Historic Saline 30% threshold of clinically significant difference Ampion is the first therapy to demonstrate significant improvement of the signs and symptoms of severe OAK patients Ampion Efficacy for
Slide 17: AP-003-C Supportive Data vs. Historical Saline Controls
Pain Function PGA AP - 003 - C Supportive Data – Ampion vs. Historical Saline Controls Reduction/Improvement in Endpoint Ampion n=144; Saline n=208. > 1.5 pain and function score for KL 4 patients required for eligibility in analysis for ‘signs and symptoms’ per FDA. Eligible saline patients across
Slide 18: Two statistically significant trials for BLA submission
AP - 003 - C and AP - 003 - A provide two statistically significant clinical trials for BLA submission that demonstrate significant effect on pain and core OAK measurements Ampion demonstrated consistent, durable pain reduction vs. saline throughout the 12 week treatment period Percent (%) Reduction
Slide 19: Consistent OARSI response across all trials
58% 60% 59% 71% 64% AP-003-A (n = 31) AP-004 (n=90) AP-003-B (n=63) AP-003-C (n=144) Combined (n=328) Ampion has consistently demonstrated significant OARSI response across all trials that exceed minimum clinically meaningful threshold • Analysis of all patients shows a 64% combined response rate, w
Slide 20: Robust safety profile with no drug-related SAEs
• 2,007 patients have participated in Ampion clinical trials (including single - injection and multiple - injection studies), and have demonstrated a robust safety profile of Ampion – No drug - related Serious Adverse Events (SAEs) – Adverse Event (AE) profile similar for saline and Ampion – Majorit
Slide 21: Open Label Extension to support expanded commercial label
An Open Label Extension (OLE) continuation of the pivotal study is underway to support an expanded commercial label with repeat administration of Ampion • Ampion is safe and well - tolerated, which has been demonstrated in all patients – Sole starting material is FDA approved HSA, which has over 60
Slide 22: Potential for disease-modifying activity
Ampio shows strong potential for disease - modifying activity through novel anti - inflammatory and immunomodulation mechanisms as well as stem cell differentiation Proinflammatory cytokines ( TNFa ) Healing - promoting prostaglandins • Ampion reduces ongoing joint damage while promoting healing – A
Slide 23: Ampion will be the first and only treatment for this unmet need
Ampion will be the first and only treatment indicated to address this unmet need x First therapy to be approved and indicated for severe OAK patients x Demonstrates clinically meaningful improvement in pain and function and quality of life x Meaningful impact improving stiffness x Lasting pain relie
Slide 24: Potential to achieve blockbuster status in the US
Given high unmet need in moderate and severe OAK, physician research suggests Ampion has the potential to achieve blockbuster status in the US References Campbell Alliance report Note: Ampion market share has been adjusted for prescribing habits by specialty (ortho, rheum, pain med), severity (mod/s
Slide 25: Mechanism may apply to other inflammatory indications
The mechanism of action of Ampion may be applied to other inflammatory indications, such as: • Other joints (hip, shoulder, wrist, hand, etc.) • Sports injuries • Systemic inflammatory conditions • Breathing disorders * Drug delivery method varies by indication Potential application in a number of o
Slide 26: Biologic exclusivity and robust IP portfolio
Ampio will receive 12 years biologic exclusivity and is also protected by a robust IP portfolio with international coverage Region Issued Patents (127) Pending Patent Applications (80) US 17 9 Ex - US 110 71 Worldwide Patent Coverage U.S. Patent Portfolio (17) 2000 2005 2010 2015 2020 2025 2030 BLA
Slide 27: Ampion manufacturing enables control over process and cost
Ampion manufacturing enables control over operational process and cost for market entry and commercialization • De - risk regulatory process – Facilitate FDA filing with direct manufacturing process experience – Facility has undergone independent inspection – Ampion process presented at Internationa
Slide 28: Executive Summary
• Ampion™ is a novel biologic set to address an unmet medical need and significant treatment gap in severe osteoarthritis of the knee (OAK) ─ FDA acknowledged there are no licensed or approved therapies to address this population • Ampion has successfully completed two pivotal Phase 3 trials for the
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