Presentation on theme: "Jefferies 2014 Global Healthcare Conference June 3, 2014."— Presentation transcript:
Jefferies 2014 Global Healthcare Conference June 3, 2014
2 Today’s Presenters Vaughan Clift, MD Chief Medical and Regulatory Affairs Officer, Ampio Pharmaceuticals, Inc. Josh Disbrow Chief Operating Officer Ampio Pharmaceuticals, Inc. President & Chief Executive Officer Luoxis Diagnostics, Inc.
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 this presentation, including statements regarding our anticipated future clinical and regulatory events, future financial position, business strategy and plans and objectives of management for future operations, are forward-looking statements. Forward looking statements are generally written in the future tense and/or are preceded by words such as “may,” “will,” “should,” “forecast,” “could,” “expect,” “suggest,” “believe,” “estimate,” “continue,” “anticipate,” “intend,” “plan,” or similar words, or the negatives of such terms or other variations on such terms or comparable terminology. Such forward-looking statements include, without limitation, statements regarding the potential future commercialization of our product candidates, the anticipated start dates, durations and completion dates, as well as the potential future results, of our ongoing and future clinical trials, the anticipated designs of our future clinical trials, anticipated future regulatory submissions and events, our anticipated future cash position and future events under our current and potential future collaborations. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including without limitation to the risks described in “Risk Factors” in Part I, Item 1A of Ampio Pharmaceuticals, Inc. Annual Report on Form 10-K and in the other reports and documents we file with the Securities and Exchange Commission from time to time. These risks are not exhaustive. Other sections of Ampio Pharmaceuticals, Inc. Annual Report on Form 10-K and such other filed reports and documents include additional factors that could adversely impact our business and financial performance. Moreover, we operate in a very competitive and rapidly changing environment. New risk factors emerge from time to time and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those projected in the forward looking statements. We assume no obligation to update or supplement forward-looking statements. 3 Safe Harbor Statement
4 Ampio Pharmaceuticals Overview Ampio Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the discovery and development of novel therapies aimed at treating common inflammatory conditions for which there are limited treatment options. We are developing compounds that serve significant medical needs in osteoarthritis, diabetic macular edema, and other prevalent, inflammatory diseases through an innovative discovery and development platform.
Ampio’s Late-Stage Pipeline Overview Ampion Phase 1Phase 2Phase 3BLA FiledApproval Osteoarthritis of the Knee (OAK) OAK Multiple Injection StudyStudy conducted in conjunction with pivotal study MOA Study in OAKStudies conducted in conjunction with pivotal study Crohn’s Disease/IBD Optina 505(b)2NDA FiledApproval Diabetic Macular Edema (DME) 5 Ampio has advanced its lead development assets into late- stage, pivotal clinical trials in the US.
6 Ampion for Osteoarthritis of the Knee (OAK) 1)D. Bar-Or, et al, Commercial human albumin preparations for clinical use are immunosuppressive in vitro, Crit Care Med 34 (2006) 1707-1712. Low molecular weight fraction of commercial human serum albumin (HSA) Aspartyl-alanyl diketopiperazine (DA-DKP) primary constituent ingredient 1 Demonstrates significant efficacy across broad spectrum of OAK patients graded Kellgren-Lawrence II through IV – radiographic OA grading instrument Established and extensive safety profile with no treatment-related adverse events Ampion is a novel biologic with a unique MOA. 3 patent families consisting of 44 issued patents and 42 pending patents Strong composition of matter claims –Pharmaceutically-acceptable carrier and DA-DKP –Filtrate produced by passing a solution of a protein or peptide over an ultrafiltration membrane with a molecular weight cutoff that retains albumin 12 years data BLA exclusivity on US upon approval & long-term supply agreement Ampion has high barriers to competitive entry.
7 New Facility: Scaling Up Production Capacity to produce 10 million+ vials/annum Easily operate at 8 million vials per annum Production lots of ~10,000 vials - 50 L HSA, 85-89% filtration yield = 10,241 to 10,723 vials/lot
8 The first Ampion pivotal SPRING Study is complete, with robust clinical improvement across a broad range of OAK patients. SPRING Study Design Summary: 9 centers, N=329, randomized 1:1:1:1, double-blind, parallel group Ampion 4 mL vs. saline 4mL & Ampion 10 mL vs. saline 10 mL Rapid completion – enrollment completed in less than 4 weeks –Study initiation date: March 29, 2013 –Study completion date: August 19, 2013 Primary efficacy endpoint: difference in pain reduction as measured by WOMAC A subscale at 12 weeks –20 week ad hoc follow-up to determine duration of effect Efficacy determined by WOMAC and PGA Safety examined as incidence and severity of AEs
Ampion demonstrated meaningful, statistically significant improvement in pain, function, and quality of life with no treatment-related AEs. 9 Patients receiving Ampion achieved: –Greater reduction in pain (WOMAC A) from baseline to 12 weeks compared to saline vehicle control (p = 0.0038) –Mean reduction in pain of 42% from baseline –Significantly greater improvement in function (WOMAC C) from baseline to 12 weeks compared to saline vehicle control (p = 0.044) –Significantly greater improvement in overall quality of life measures (Patient Global Assessment) from baseline to 12 weeks compared to saline vehicle control (p = 0.012) Clinical efficacy was observed as early as four weeks and sustained through 20 weeks in subset analysis –4 mL Ampion dose selected for second pivotal study –WOMAC A mean difference from saline control (p=0.025) Ampion was well tolerated with minimal adverse events and no drug-related serious adverse events
10 Second Ampion Pivotal Trial: STEP Study STEP Study Summary: 17 centers, N=538, randomized 1:1, double-blind, parallel group Power to demonstrate a significant treatment effect is 90% Ampion 4 mL vs. saline 4mL Stratified by KL 2 (~25%) and KL 3 / 4 Rapid completion – enrollment completed in less than 2 months Primary efficacy endpoint: difference in pain reduction as measured by WOMAC A subscale at 12 weeks 20 week follow-up to determine duration of effect Efficacy determined by WOMAC and PGA Safety examined as incidence and severity of AEs
11 STEP Study: Milestones at a Glance First Patient First Visit: January 9 th, 2014 Last Patient First Visit: February 21 st, 2014 Last Patient Last Visit (12 weeks): ~May 22 nd 2014 Last Patient Last Visit (20 weeks): ~July 17, 2014 Data Readout: Q3 2014 BLA Submission to Follow
12 Optina (ultra low-dose danazol) 1) Thomas GW, et al. Biphasic effect of danazol on human vascular endothelial cell permeability and f-actin cytoskeleton dynamics. Biochem Biophys Res Commun. 2012;421:707-712 Optina is a fat soluble low molecular weight weak androgen that affects vascular permeability through the endothelial barrier in a biphasic manner 1 Inhibits vascular permeability by stimulating formation of cortical actin ring and binding molecules 1 Demonstrated improvement in central retinal thickness in initial clinical trial, with strong treatment signal observed in current OPTIMEYES Study Optina has a demonstrated clinical effect in diabetic macular edema. 2 patent families consisting of 40 issued patents and 44 pending patents Newly discovered mechanism of action enables broad intellectual property position –Angiogenesis and vascular permeability are distinct phenomena –It is surprising and unexpected that danazol affects vascular permeability at low doses and that the dose of danazol required for treating vascular permeability is different from the dose required for treatment of angiogenesis Optina has strong IP protection with high barriers to entry.
13 OptimEyes Study Summary: AP-05-002 AP-05-002 Study Design Summary: 22 centers, N=450, randomized 1:1:1, double-masked Total Enrolled: 347 subjects (as of 5/28/2014) Optina (low-dose danazol) oral capsule, 1 mg per BMI, or 0.5 mg per BMI and placebo divided into two daily doses Primary efficacy endpoint: Change in BCVA letters read from baseline to 12 weeks Safety examined as incidence and severity of adverse events and glycemic, liver, renal, lipid, and hormone assays.
14 An Independent Data Review Committee conducted an interim analysis of the OPTIMEYES Study – with treatment effect observed Independent Data Review Committee (IDRC) Summary Findings: At least thirty percent (30%) of patients had reached the first 4 week time point and their clinical results; considered representative data for the trial. Analysis parameters were defined a priori and could have resulted in three possible scenarios as follows: –Termination of the study due to lack of efficacy or safety concerns –Delayed analysis if no conclusion could be drawn –Indication of an optimum dose if sufficient differentiation from placebo was present to indicate clinical benefit and no serious adverse events “After a thorough review of the interim data from the ongoing study, it was determined that there was a treatment dosage that was demonstrating a potentially beneficial anatomic effect. Given that there were no significant safety concerns identified in the study to date, a recommendation to continue the trial was made” OPTIMEYES Study IDRC, September 2013
16 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 exist to measure oxidative stress. Oxidation-reduction potential is a measure of homeostasis detecting the balance between antioxidants and pro-oxidants in the body. –A complete picture of oxidative stress
17 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 certification awarded January 2014 –Research partnerships in place with major research centers throughout the world Luoxis is moving rapidly toward commercialization. –CE Mark obtained in Q1 2014 –Confidential EU-specific partnering discussions underway –First pharma research agreement signed in Q2 2014 –FDA submission expected in 2014 as 510k de novo
18 Luoxis-specific milestones since Ampio carve out (Private placement closed May 2013) Oct-2013: Clinical validation study in TBI; issuance of patent family covering broad range of clinical applications Dec-2013: FDA confirms 510(k) de novo classification is appropriate for RedoxSYS 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 Jun-2014: Report top-line results for ORP in stroke Jul-2014: Collaboration with first independent U.S. academic institution
19 3-Pronged Approach for Broad Acceptance Continue to Drive Clinical Validation Pursue Global Regulatory Approvals Expand Pharma Relationships
20 Vyrix Pharmaceuticals is advancing Zertane toward FDA approval and commercialization. Vyrix was created in November 2013 to increase the value of the sexual dysfunction asset portfolio. –Zertane submitted to Australian Therapeutic Goods Administration (TGA) with approval expected in 2014. –TGA clearance enables submissions and near-term commercialization in numerous countries –Multiple ex-US partnerships underway Vyrix expects to initiate pivotal US trials in the near-term. –Clearly defined pathway with two pivotal trials already completed outside US –Primary endpoints validated and agreed upon with FDA –Pivotal trial protocol finalized; IND submission pending
Ampion is positioned for a near-term BLA submission 21 Ampio Pharmaceuticals Summary Novel biologic demonstrating positive pivotal data with final Phase III top-line data expected 3Q 2014 Significant efficacy demonstrated in moderate to severe OAK patients Ampio has a portfolio of innovative late-stage assets Positive interim results confirmed observed treatment effects through IDRC Phase IIb top-line data expected 3Q 2014 Optina has demonstrated a treatment effect in DME Lead products address underserved, large markets and strong intellectual property Well-defined, efficient clinical development plans with late-stage trials underway Ampio’s subsidiaries are creating new value Luoxis has a fully developed asset that is nearing commercialization. Vyrix is progressing Zertane into pivotal US trials with approvals pending outside the US.
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