Ampio Pharmaceuticals, Inc. - FORM 8-K - EX-99.1

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EX-99.2 - EX-99.2 - Ampio Pharmaceuticals, Inc.	d792397dex992.htm

Shareholder Presentation

September 2014

Exhibit 99.1

Agenda

Topic

Speaker

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

Michael Macaluso

Formal Business

2014 Annual Meeting of Shareholders

Formal Business

2014 Annual Meeting of Shareholders

Corporate Presentation

Formal Business

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 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.

Scientific Address

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:

Ampion™: Overview of Anti-inflammatory and

Healing Effects

Ampion™: Cortical Actin Ring Formation in

Endothelial Cells

Saline

Ampion™

Ampion™: Filopodia Formation in Stem Cells

Saline

Ampion™

Ampion™: Micromass Condensation (Cartilage

Formation)

Saline

DADKP

Ampion™

December 2013

Optina™: OCT in Open-label Extension in Patient A

Beginning of OLE

+3 Months

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

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)

Product Overview

What is a BLA?

BLA FILING

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

Labeling

•

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 are of rendering of new facility, not of actual facility.

Product/Manufacturing

Facility tour after the meeting today

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 by Ampio, and outsourced, will be brought

back in-house for commercial scale manufacture

Engineering runs to support scale-up to commercial

manufacture are underway

Registration batches for BLA have been scheduled to begin after

all validation activity is complete

Clinical Summary: Ampion ™

“Two adequate and well-controlled trials with

reproducible evidence of efficacy are required to

support approval of a novel product for…the

management of the pain of OA…[T]here is

one primary endpoint,

either a pain score such

as the NRS or the WOMAC pain subscale”

FDA

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, double-blind study to evaluate the efficacy

and safety of two doses of intra-articular injection of Ampion™

in adults with

pain due to osteoarthritis of the knee.

“Upon review of the dataset

received on November 5,

2013, FDA concludes that

study AP-003-A can be

considered as one of two

“pivotal trials”

required in

support of a BLA.”

FDA

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

Due to the strong results at Week 12, the study was amended and an

optional follow up visit at Week 20 was added

ANCOVA, analysis of covariance; K-L, Kellgren-Lawrence; LS, least squares; WOMAC, Western Ontario and McMaster

Universities

Osteoarthritis.

Repeated measures ANCOVA over 20 weeks, adjusted for baseline values.

P=0.005

P=0.04

SPRING Study Significant Pain Relief Over 20 Weeks

in K-L Grades 3 and 4

-0.99

-0.85

-0.65

-0.58

-1.2

-1

-0.8

-0.6

-0.4

-0.2

WOMAC A pain

WOMAC C function

LMWF-5A (n=36)

Control (n=28)

Clinical Summary: Ampion™

“FDA recommends that Ampio conduct an adequately

powered study to confirm the effect size of AP-003-A and

design the study to obtain information on the duration of

action and repeat dosing.”

FDA

Adequately powered: 90%

Duration of action: 20 Weeks

Repeat dosing: Post-Approval

Clinical Summary: Ampion™

A prospective phase I/II study to evaluate the safety and exploratory efficacy of three intra-

articular

injection

Ampion™

mL)

administered

two

weeks

part

adults

with pain due

to osteoarthritis of the knee.

Single center

Phase 1: Open label (N=7)

Phase II: Randomized (N=30*)

Primary endpoint: 20 Weeks

Study duration: 52 Weeks

Exploration of additional clinical benefits

High resolution MRI analysis

Knee aspirations to assess synovial fluid

Serum biomarkers

Activity and exercise logs

Investigating Healing

*study is being updated to N=40

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 controlled’: Normal saline

Primary endpoint: Change in WOMAC score at 20 weeks

Duration of action: Study extends over 24 weeks

Repeat dosing: 3 injections (Baseline, Week 2, Week 4)

SECOND PIVOTAL TRIAL: Stride Study

Clinical Summary: Ampion™

“FDA recommends that Ampio conduct an adequately

powered study

to confirm the effect size of AP-003-A

and design the study to obtain information on the

duration of action

and repeat dosing.”

FDA

Adequately powered: 90%

Duration of action: 20 Weeks

Repeat dosing: Post-Approval

Adequately powered: 90%

Duration of action: 24 Weeks

Repeat dosing: 3 Doses

*Underline emphasis added to quotation for clarification and discussion

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

355 patients

randomized

0.5 mg per BMI

4 week interim analysis

Announced 4Q 2013

1.0 mg per BMI

Placebo

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 committee identified optimal dosage

demonstrating potentially beneficial anatomic and clinical effect

•

Enrollment complete. Closed enrollment Q3 2014 with top line results expected Q4

2014

•

Optina 505(b)(2) designation enables efficient path toward NDA filing

Product Portfolio: Optina™

A randomized, placebo-controlled, double-masked study to evaluate the efficacy and

safety of two doses of oral Optina™

in adult patients with diabetic macular edema.

Regulatory Review

Ampio’s Pipeline Overview

Ampion™

Phase 1

Phase 2

Phase 3

BLA

Filed

Approval

Degenerative Joint Diseases

Osteoarthritis of the Knee (OAK)

T-Cell Mediated Disease

Inflammatory Bowel Disease

Core Development Assets

Optina™

Phase 1

Phase 2/Phase 3

NDA

Filed

Approval

Diabetic Angiopathies

Diabetic Macular Edema (DME)

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 person by or for

whom the investigations were conducted" (21 U.S.C.355(b)(2)).

505(b)(2) Regulatory Pathway

Relatively LOW RISK

because of existing safety and efficacy information

LOWER COST

due to the smaller scope and number of potential studies

INCREASED SPEED

due to fewer studies

RAPID REGULATORY PATH

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 from Baseline to

12 weeks

•

Safety examined as incidence and severity of adverse events and

glycemic, liver, renal, lipid, and hormone assays.

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 steroids and HA.

Ampion™

NSAIDs, COX-2 Inhibitors, IA Steroids, HA

Surgery

Arthroscopic,

osteotomy, total or

partial knee

arthroplasty, cartilage

grafting¹

Severe

Large osteophytes,

narrowing of joint space,

severe sclerosis and

deformity of bone ends¹

Moderate

Multiple osteophytes

and possible narrowing

of joint space¹

Mild

Osteophyte

development and

possible narrowing of

the joint space¹

Hillary J. Braun and Garry E. Gold. Diagnosis of osteoarthritis: Imaging. Elsevier November 2011. 2. www.aaos.org “Arthritis of the knee” Accessed October 6, 2013.

Relationship between patient-reported disease severity in osteoarthritis and self-reported pain, function and work productivity. Sadosky et al. Arthritis Research & Therapy 2012.

Osteoarthritis Epidemiology Report. DataMonitor, Inc. 2013.

Treatment of Osteoarthritis of the Knee, Evidence-Based Guideline 2nd Edition. American Academy of Orthopaedic Surgeons. May 18, 2013.

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) should be

packaged to prevent

contamination and

unacceptable

deterioration during

transport and

storage.

“…Distribution Department will prepare each

shipment using pre-qualified insulated

shipping containers and gel packs…Shippers

are

pre-qualified

maintain

15°–

25°C

for 48 hours…”

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.

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 group

•

2014:

Initiation

operations

Ampion™

manufacturing

facility

•

Q3 2014: Pivotal STEP Study completed

–

Logistical issue identified related to shipping conditions of study drug

•

Q3 2014: Pivotal multiple injection (MI) study initiated

–

86% improvement in pain from baseline (6 week time point)

•

Q1/Q2

2015:

Expected

results

and

filing

Ampion™

BLA

2013

2014

Q1/

2015

Doctor’s Thoughts

62 yr old female; received Ampion™

in the SPRING study:

“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

trial and although the actual injection really hurt, I now feel like a

new person. I can walk up the stairs and am able to do so many

things I was previously unable to do. I can’t remember the last time

I have had to take pain medication for my knee. I am thrilled. I

want to walk over to my orthopedic doctor and show him how well

I am doing now.

Just today, I found that I was on the real drug (Ampion™). It was

something I had long suspected since I had been having such

improvement in function. For some time, I have wished I could have

an injection in my other knee as well.”

–

Patient 04-050

Doctor’s Thoughts and Patient Testimonials

•

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

Placebo

4 mL

Ampion™

4 mL

Placebo

4 mL +

10 mL

Ampion™

4 mL +

10 mL

(N=83)

(N=164)

(N=165)

Any Related AE

13 (16%)

9 (11%)

21 (13%)

17 (10%)

Any Related Serious AE

0 (0%)

Any AE Resulting in Treatment Change

0 (0%)

Any AE Resulting in Study

Discontinuation

0 (0%)

Any Serious AE Resulting in Death

0 (0%)

Inherent, Established Safety of Ampion™

Doctor’s Thoughts

”The effects of Ampion™

are truly remarkable. I have never

seen an injectable drug into the knee with this degree of pain

relief and improvement of function. This may change the way

we treat patients with osteoarthritis of the knee”.

Dr. John Schwappach, orthopedic surgeon and the Principal

Investigator in the Ampion™

multiple injections trial

Luoxis Diagnostics,

subsidiary of

Ampio Pharmaceuticals

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

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

–

Market development underway through research partnerships at

major research centers throughout the world

•

Luoxis is moving rapidly toward commercialization with

market development underway

–

CE Mark obtained in Q1 2014

–

Distribution partnering discussions underway in Europe

–

Distribution partnership discussions underway in Japan

–

First pharma research agreement signed in Q2 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 major academic research

center

Jul-2014: Collaboration with first independent U.S. academic institution

Sept-2014: Presented TBI clinical study data at AAST (American Association for

the Surgery of Trauma)

Q4 2014-Q1 2015: Anticipate first distribution agreements signed in Europe and

Japan for introduction into research market and development of the clinical

market

Q4 2014: Expect additional pharma research agreements initiated

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

Approvals

Expand

Pharma

Relationships

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

–

Research partnerships in place with major research centers

throughout the world

•

Luoxis is moving rapidly toward commercialization.

–

CE Mark in Q1 2014

–

Health Canada clearance in Q2 2014

–

Launching into research market for clinical market development

–

FDA submission expected in 2014 as 510k de novo

–

Confidential commercial partnering discussions actively underway

Company Updates and

Q&A

Headquarters:

373 Inverness Parkway, Suite 200

Englewood, CO 80112

Ampio Pharmaceuticals, Inc. - FORM 8-K - EX-99.1 - September 19, 2014

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Ampio Pharmaceuticals, Inc. Reports