Ampio Pharmaceuticals Initiates Clinical Trial of Optina™ in Diabetic Macular Edema (DME): First Patient Dosed

Ampio Pharmaceuticals Initiates Clinical Trial of Optina™ in Diabetic Macular
                       Edema (DME): First Patient Dosed

PR Newswire

GREENWOOD VILLAGE, Colo., Feb. 27, 2013

GREENWOOD VILLAGE, Colo., Feb. 27, 2013 /PRNewswire/ --Ampio Pharmaceuticals,
Inc. (NASDAQ: AMPE) announced oral dosing of the first patient in a 505(b)(2)
clinical trial of the investigational drug Optina in diabetic macular edema.
The trial will evaluate Optina™ in adults with recent onset diabetic macular
edema. Last year, the FDA granted Optina™ 505(b)(2) status and drugs
designated under this pathway can be approved on a single trial.

(Logo: http://photos.prnewswire.com/prnh/20120516/MM09116LOGO)

Michael Macaluso, Chairman and CEO of Ampio, commented "We dosed the first
patient with oral Optina™' today. We believe that Optina has the potential to
successfully treat patients afflicted with diabetic macular edema and satisfy
a large unmet clinical need with a successful oral therapy. Based on the
encouraging results from the Canadian study, we are very excited about
Optina™'s potential to allow diabetic patients to avoid direct injections into
the eye, a requirement of all other treatment regimens."

The multicenter trial is designed to evaluate the safety and efficacy of oral
Optina™ compared with placebo given over a period of 12 weeks in adult
patients with DME. A total of 450 patients are expected to enroll. Patients
are randomized to receive one of two oral doses of Optina™ (0.5mg per BMI and
1.0mg per BMI per day) or placebo. After patients have completed 4 weeks of
initial treatment, an interim analysis will occur to determine the best dose
of Optina™. Following the 12 week active treatment period, there will be a
further 4 week washout period to determine regression of treatment effect. The
primary endpoint is improvement in visual acuity (VA), defined by responder
status, compared to placebo. Secondary endpoints are 1) measurements of
changes in VA and central macular thickness (CMT) in treated patients compared
to placebo and, 2) safety and tolerability of the two Optina™ doses. Following
treatment and washout, patients will be assessed for vision regression and a
12 week open label extension study will be offered to evaluate the duration of
effect of the optimal dose.

About Optina™
Optina™ is a drug based on a low dose of the weak androgen,
low-molecular-weight, very lipophilic steroid danazol. Oral administration of
low dose danazol to patients with DME is safe with no evidence of serious
adverse events. Ampio's in vitro data suggest that danazol has a biphasic
effect on endothelial cells: At low doses, danazol decreases vascular leakage,
while at higher concentrations an increase in vascular permeability is
observed. This biphasic effect was supported by the efficacy of danazol in
vivo at various BMIs. From Ampio's previously announced results, Optina™
appears to reduce DME in a BMI dosage-adjusted manner and appears to trend
toward improved visual acuity and seems to be safe with few, if any, side
effects.^1, 2

About Diabetic Macular Edema
Diabetic Macular Edema (DME) is a swelling of the retina indiabeticpatients
due to leaking of fluid from blood vessels within the macula. The macula is
the part of the eye responsible for sharp central vision. There are
approximately 26 million people in the United States with diabetes and DME can
occur in both Type 1 and Type 2 diabetes. Up to 10% of diabetics will develop
DME during their lifetime and up to 75,000 new cases of DME are estimated to
occur each year in the United States^3. Current treatments include anti-VEGF
drugs, corticosteroid-based regiments, and laser surgery that helps seal the
leaky blood vessels. Drug delivery by injection into the vitreous of the eye
is a limitation of these new treatments because of the potential complications
due to repeated injections into the eye such as infection, pain, hemorrhage
and increased intraocular pressure.

About Ampio Pharmaceuticals
Ampio Pharmaceuticals, Inc. is a biopharmaceutical company focused on the
rapid development of therapies to treat prevalent inflammatory conditions for
which there are limited treatment options. It aims to provide medicines to
improve the health and quality of life of patients with minimal side effects.
Ampio is developing compounds that decrease inflammation by 1) inhibition of
specific pro-inflammatory compounds by affecting specific pathways at the
protein expression and at the transcription level or 2) activation of a
specific phosphatase or depletion of the available phosphate needed for the
inflammation process. For more information visit: www.ampiopharma.com.

Forward Looking Statement
This press release includes forward-looking statements within the meaning of
Section27A of the Securities Act of 1933, as amended, and Section21E of the
Securities Exchange Act of 1934, or the Exchange Act. All statements other
than statements of historical facts contained in this press release, 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 the risks described in "Risk Factors" in PartI, Item1A of
Ampio's Annual Report. These risks are not exhaustive. Other sections of this
Annual Report 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

References
1. Bar-Or D, Thomas GW, Salottolo K, et al. Oral Danazol for DME. Retina
Today. 2012; Vol. 7, No. 7: 68-70. Available at:
http://bmctoday.net/retinatoday/2012/10/article.asp?f=oral-danazol-for-dme
2. Thomas GW, Rael LT, Bar-Or R, 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. Available at:
http://www.ncbi.nlm.nih.gov/pubmed/22542943
3. Ali, F.A. A review of diabetic macular edema. Digital Journal of
Ophthalmology, vol. 3, no. 6, 1997. Available at:
http://www.djo.harvard.edu/site.php?url=/physicians/oa/387

SOURCE Ampio Pharmaceuticals, Inc.

Website: http://www.ampiopharma.com
Contact: Rick Giles, Director of Investor Relations, Ampio Pharmaceuticals,
Inc., +1-720-437-6530, rgiles@ampiopharma.com
 
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