47 slides from Ampio Pharmaceuticals's shareholder meeting presentation, Sep 22, 2014
Slide 1: Shareholder Presentation
Shareholder Presentation September 2014 Exhibit 99.1
Slide 2: Agenda
Topic Speaker Agenda Formal Business Greg Gould Scientific Address David Bar-Or, MD Product Overview Holli Loose, MsC Clinical Review Vaughan Clift, MD Doctor’s Thoughts John Ervin, MD Doctor’s Thoughts John Schwappach, MD Luoxis Overview Josh Disbrow, MBA Company Updates Michael Macaluso Q&A Michae
Slide 3: Formal Business
Formal Business 2014 Annual Meeting of Shareholders Formal Business
Slide 4: 2014 Annual Meeting of Shareholders
2014 Annual Meeting of Shareholders Corporate Presentation Formal Business
Slide 5: Safe Harbor Statement
Safe Harbor Statement This presentation includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, or the Exchange Act. All statements other than statements of historical facts contained in
Slide 6: Scientific Address
Scientific Address
Slide 7: Lead Products
Lead Products Solid science comprising over 30 years of research Ampion™ 50 issued patents worldwide 118 pending patents worldwide Optina™ 83 issued patents worldwide 81 pending patents worldwide Safe & Effective Patent Portfolio: Only orally available treatment for DME Patent Portfolio:
Slide 8: Ampion: Overview of Anti-inflammatory and Healing Effects
Ampion™: Overview of Anti-inflammatory and Healing Effects
Slide 9: Ampion: Cortical Actin Ring Formation in Endothelial Cells
Ampion™: Cortical Actin Ring Formation in Endothelial Cells Saline Ampion™
Slide 10: Ampion: Filopodia Formation in Stem Cells
Ampion™: Filopodia Formation in Stem Cells Saline Ampion™
Slide 11: Ampion: Micromass Condensation (Cartilage Formation)
Ampion™: Micromass Condensation (Cartilage Formation) Saline DADKP Ampion™
Slide 12: Optina: OCT in Open-label Extension in Patient A
December 2013 Optina™: OCT in Open-label Extension in Patient A Beginning of OLE +3 Months
Slide 13: Optina: Patient B
Optina™: Patient B Kenalog intravitreal 8/22/06 PRP laser 8/28/04, 3/24/05, 6/20/07 Focal laser 8/20/12, 12/10/12 Avastin 10/1/12, 11/26/12, 4/14/13
Slide 14: Optina: VA and OCT in Patient B
Optina™: VA and OCT in Patient B OCT=437u BCVA OD 20/50 ETDRS (62) (37days no drug) OCT=246u BCVA OD 20/50 ETDRS (67)
Slide 15: Product Overview
Product Overview
Slide 16: BLA Filing
BLA FILING What is a BLA? A Biologics License Application (BLA) is a request for permission to introduce a biologic product into interstate commerce (21 CFR 601.2). Requirements for a BLA include: o Applicant information o Product/Manufacturing information o Pre-clinical studies o Clinical studies o
Slide 17: Product/Manufacturing Facility
• Facility will have an annual production capacity of ~10 million vials of Ampion • Source material, human serum albumin (HSA), required to meet this capacity has already been secured through a long-term, non- exclusive agreement • cGMP compliant – US and European regulatory compliant Note: Pictures
Slide 18: Product/Manufacturing
Product/Manufacturing FDA has issued a written response accepting the technical transfer, analytical, scale-up and manufacturing plan for production of Ampion™ in the new facility. Technical transfer from contract manufacturing to commercial facility in Englewood, CO is underway Analysis developed b
Slide 19: First Pivotal Trial: SPRING Study
FIRST PIVITOL TRIAL: SPRING Study FIRST PIVITOL TRIAL: SPRING Study Clinical Summary: Ampion™ ‘Adequate’ (n=329 across 9 sites) ‘Well controlled’ (normal saline) Trial was ‘well conducted’ The study met its primary endpoint (change in WOMAC score at 12 weeks). A randomized, placebo-controlled, doubl
Slide 20: Clinical Summary: Ampion
Clinical Summary: Ampion™ -1.1 -1 -0.9 -0.8 -0.7 -0.6 -0.5 -0.4 -0.3 -0.2 -0.1 Study Week Effects of 4mL Ampion™ treatment on WOMAC pain over time
Slide 21: SPRING Study Significant Pain Relief Over 20 Weeks in K-L Grades 3 and 4
Due to the strong results at Week 12, the study was amended and an Due to the strong results at Week 12, the study was amended and an optional follow up visit at Week 20 was added optional follow up visit at Week 20 was added ANCOVA, analysis of covariance; K-L, Kellgren-Lawrence; LS, least squares;
Slide 22: Clinical Summary: Ampion
Clinical Summary: Ampion™ “FDA recommends that Ampio conduct an adequately “FDA recommends that Ampio conduct an adequately powered study to confirm the effect size of AP-003-A and powered study to confirm the effect size of AP-003-A and design the study to obtain information on the duration of desi
Slide 23: Clinical Summary: Ampion
Clinical Summary: Ampion™ A prospective phase I/II study to evaluate the safety and exploratory efficacy of three intra- articular injection of Ampion™ (4 mL) administered two weeks a part in adults with pain due to osteoarthritis of the knee. Single center Phase 1: Open label (N=7) Phase II: Random
Slide 24: Second Pivotal Trial: Stride Study
Clinical Summary: Ampion™ A randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of three intra-articular injections of Ampion (4 mL) administered two weeks apart in adults with pain due to osteoarthritis of the knee. ‘Adequate’: N = 320 across 14 sites ‘Well contro
Slide 25: Clinical Summary: Ampion
Clinical Summary: Ampion™ “FDA recommends that Ampio conduct an adequately “FDA recommends that Ampio conduct an adequately powered study powered study to confirm the effect size of AP-003-A to confirm the effect size of AP-003-A and design the study to obtain information on the and design the study
Slide 26: R&D: Ampion
R&D: Ampion™ Investigations into additional uses for Ampion ™ Crohn’s disease – investigation into multiple uses and indications for Ampion™ o Oral formulation that can be made in our facility in Englewood, CO o Proof of Concept study was accepted by FDA
Slide 27: Product Portfolio: Optina
355 patients randomized 0.5 mg per BMI 4 week interim analysis Announced 4Q 2013 1.0 mg per BMI Placebo Washout Washout 12 week analysis Expected Q4 2014 12 Week Open Label Extension • ~50% of patients are treatment naïve; ~50% have received anti-VEGF in past • Interim Analysis by independent commit
Slide 28: Regulatory Review
Regulatory Review
Slide 29: Ampio's Pipeline Overview
Ampion™ PC Phase 1 Phase 2 Phase 3 BLA Filed Approval Core Development Assets Ampio’s Pipeline Overview Degenerative Joint Diseases Osteoarthritis of the Knee (OAK) T-Cell Mediated Disease Inflammatory Bowel Disease Optina™ PC Phase 1 Phase 2/Phase 3 NDA Filed Approval Diabetic Angiopathies Diabetic
Slide 30: 505(b)(2) Regulatory Pathway
505(b)(2) Regulatory Pathway Product Portfolio: Optina™ A 505(b)(2) application is one for which one or more of the investigations relied upon by the applicant for approval "were not conducted by or for the applicant and for which the applicant has not obtained a right of reference or use from the p
Slide 31: Optina
Optina™ Sub-Populations • 22 centers, N=355, randomized 1:1:1, double-masked • Total Enrolled: 359 subjects; 432 study eyes • ~50% of patients were refractory to anti-VEGEF intra-ocular therapy • Proportion of patients with renal impairment • Primary efficacy endpoint: Change in BCVA letters read fr
Slide 32: Ampion addresses a clear medical need in osteoarthritis of the knee
Ampion™ addresses a clear medical need in the treatment of Osteoarthritis of the Knee. • 60% of OAK patients suffer from moderate or severe disease. ² • Approximately 27 million OAK patients in the US are symptomatic. ³ • Latest AAOS clinical practice guidelines recommend against the use of IA stero
Slide 33: Clinical Summary: Hurdles
Clinical Summary: Hurdles A randomized, placebo-controlled, double-blind study to evaluate the efficacy and safety of a single 4 mL intra-articular injection of Ampion ™ in adults with pain due to osteoarthritis of the knee. Per FDA Good Clinical Practice 5.13.3: The investigational product(s) shoul
Slide 34: Clinical Summary: Solutions
Clinical Summary: Solutions A randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of three intra-articular injections of Ampion ™ (4 mL) administered two weeks apart in adults with pain due to osteoarthritis of the knee.
Slide 35: Ampion Clinical Development Progress & Expected Milestones
Ampion™ Clinical Development Progress & Expected Milestones • Q3 2013: Positive results reported from pivotal SPRING Study showing: – 42% reduction in pain from baseline (12 week endpoint) – Pronounced effect in KL 3 and 4 patients with no serious drug-related adverse events across entire treatment
Slide 36: Doctor's Thoughts
Doctor’s Thoughts
Slide 37: Patient testimonial from the SPRING study
62 yr old female; received Ampion ™ in the SPRING study: “I talked with a friend from work and he mentioned Dr. Ervin and “I talked with a friend from work and he mentioned Dr. Ervin and the clinical trials that he runs at his office. I enrolled in the Ampion the clinical trials that he runs at his
Slide 38: Inherent, Established Safety of Ampion
Placebo 4 mL Ampion™ 4 mL Placebo 4 mL + 10 mL Ampion™ 4 mL + 10 mL • Ampion™ is the low molecular weight fraction of FDA approved / regulated Human Serum Albumin (HSA) • HSA has its own approved safety record • In the AP-003-A trial there were no drug-related serious adverse events (N=83) (N=83) (N
Slide 39: Doctor's Thoughts
Doctor’s Thoughts ”The effects of Ampion™ ”The effects of Ampion™ are truly remarkable. I have never are truly remarkable. I have never seen an injectable drug into the knee with this degree of pain seen an injectable drug into the knee with this degree of pain relief and improvement of function. Th
Slide 40: Luoxis Diagnostics, subsidiary of Ampio Pharmaceuticals
Luoxis Diagnostics, subsidiary of Ampio Pharmaceuticals
Slide 41: Oxidation-reduction potential: A 'vital sign'
Oxidation-reduction potential: A ‘vital sign’ assessing the body’s response to oxidative stress • Oxidative stress is the body’s response to physiologic stress brought on by a depletion of natural antioxidants in response to: – Illness – Injury – Inflammation • While well recognized, no good methods
Slide 42: Luoxis Diagnostics has significantly advanced development of the ORP device platform
Luoxis Diagnostics has significantly advanced development of the oxidation-reduction potential (ORP) device platform. • Luoxis has a fully-developed IVD asset. – Clinical validation studies completed in traumatic brain injury (TBI), multi-trauma, hip fracture, and stroke – ISO 13485:2003 certificati
Slide 43: Corporate milestones since Jan 2014
Corporate milestones since Jan 2014 Jan-2014: ISO 13485:2003 medical device certification Apr-2014: CE Marking/Health Canada approval Apr-2014: First clinical development project with Big Pharma May-2014: First instrument placed with pharma company June-2014: First instrument placed in Europe with m
Slide 44: 3-Pronged Approach for Broad Acceptance
3-Pronged Approach for Broad Acceptance Nearly 50 studies underway or in planning stages Two large Phase 2b studies underway utilizing ORP, working with 5 pharma companies CE Mark enables global market clearances; FDA studies underway Continue to Drive Clinical Validation Pursue Global Regulatory Ap
Slide 45: Luoxis Diagnostics has significantly advanced development of the ORP device platform
Luoxis Diagnostics has significantly advanced development of the oxidation-reduction potential (ORP) device platform. • Luoxis has a fully-developed IVD asset. – Clinical validation studies completed in traumatic brain injury and multi-trauma – ISO 13485:2003 certification awarded January 2014 – Res
Slide 46: Company Updates and Q&A
Company Updates and Q&A
Slide 47: Headquarters, Englewood, CO
Headquarters: 373 Inverness Parkway, Suite 200 Englewood, CO 80112 www.ampiopharma.com