Ampio Pharmaceuticals, Inc. Announces Positive Results from Independent Panel
Review of the Interim Analysis of the Optina(TM) Trial for Diabetic Macular
GREENWOOD VILLAGE, Colo., Oct. 7, 2013
GREENWOOD VILLAGE, Colo., Oct.7, 2013 /PRNewswire/ --Ampio Pharmaceuticals,
Inc. (NYSE MKT: AMPE) today announced interim results from the ongoing 450
patient, dose finding, 505 (b) 2 study of Optina^TM as a treatment for
Diabetic Macular Edema (DME). This interim analysis was conducted by an
Independent Data Review Committee (IDRC) comprised of a statistician and an
ophthalmologist/retinologist, who were permitted to view the unmasked data
from the trial. At least thirty percent (30%) of patients had reached the
first 4 week time point and their clinical results were considered
representative data for the trial.
Dr. Vaughan Clift, Ampio's Chief Regulatory Officer explained: "As is common
with interim analyses of this sort, the analysis parameters were defined a
priori and could have resulted in three possible scenarios as follows: 1)
Termination of the study due to lack of efficacy or safety concerns, 2)
delayed analysis if no conclusion could be drawn or 3) indication of an
optimum dose if sufficient differentiation from placebo was present to
indicate clinical benefit and no serious adverse events. The IDRC elected the
third option and stated:
"After a thorough review of the interim data from the ongoing study, it was
determined that there was a treatmentdosage that was demonstrating a
potentially beneficial anatomic effect. Given that therewere no
significantsafety concerns identified in the study to date, a recommendation
to continue the trial was made".
Dr. Clift further explained "This IDRC decision will allow for the
continuation and completion of the trial and, importantly, the immediate
initiation of an open label extension study using this optimum dose of
Optina^TM for all patients who have completed the trial and wish to continue
treatment for an additional 12 weeks. Patients who received either dose of
Optina^TM in the masked trial as well as those who received placebo will be
eligible for inclusion in this Open Label Extension of the Trial.
"We are very pleased with both the IDRC recommendation and the rapid patient
enrollment, which now exceeds 250 patients. We expect the trial to be
completed in the first quarter of 2014, and anticipate releasing top-line
results as soon as they become available to Ampio. FDA has agreed to the 505
(b) 2 pathway for this drug which, given no significant safety concerns, could
allow approval based on this single clinical trial."
About the Optina Trial
Patient enrollment for the 450 patient US trial began in February 2013 and
over 250 patients have been enrolled to date. The multicenter, dose-finding
trial is designed to evaluate the safety and efficacy of oral Optina^TM
compared with placebo over 12 weeks in adult patients with DME. Patients are
randomized (1:1:1)to receive one of two oral doses of Optina^TM (0.5mg per
BMI and 1.0mgper BMI per day) or placebo and are treated for 12 weeks. The
primary endpoint of the Phase IIb trial is improvement in best-corrected
visual acuity (BCVA) in treated patients compared to placebo at 12 weeks.
Secondary endpoints are (i)measurement of changes in central macular
thickness in treated patients compared to a placebo and (ii)safety and
tolerability of the two Optina doses.
About Open Label Extension of
Following the active treatment period, all patients will be followed for four
weeks without treatment in order to study any regression and then immediately
allowed the option to enter into a 12 week open label extension of the trial
in which they will receive the optimal dose of Optina^TM as determined by this
interim analysis after which time their improvement in BCVA will be evaluated.
Optina^TM is a low-dose formulation of danazol that we are developing to treat
diabetic macular edema. Danazol is a synthetic derivative of modified
testosterone ethisterone, and we believe it affects vascular endothelial cell
linkage in a biphasic manner. At low doses, danazol decreases vascular
permeability by increasing the barrier function of endothelial cells. The
lipophilic low-molecular-weight weak androgen has the potential to treat
About Diabetic Macular Edema
Type 1 and type 2 diabetes mellitus affects 26million people in the United
States. One of the many symptoms of diabetes is the local and systemic
inflammation of the microvascular system. Diabetic retinopathy is a
complication of diabetes and is characterized by damage to the blood vessels
of the retina and can either be proliferative or non-proliferative.
Proliferative damage occurs when a reduction in oxygen levels in the retina
due to impaired glucose metabolism causes fragile blood vessels to grow in the
vitreous humor. Non-proliferative damage occurs when existing vessels
experience poor endothelial cell linkage due to increased blood glucose levels
and hypertension. Macular edema is the most common form of non-proliferative
diabetic retinopathy. In diabetic macular edema, prolonged hyperglycemia
compromises endothelial cell linkage leading to vascular permeability. The
leakage of fluid, solutes, proteins and immune cells cause the macula to swell
and thicken. This leads to damage of the central retinal tissue and can
significantly impair sharp central vision. The prevalence of diabetes is 11.3%
of the population above the age of 20, with an annual incidence of 1.9million
cases in the United States alone. In this population, the prevalence of
diabetic macular edema is estimated at 30% of patients inflicted by the
disease for 20 years or more.
About Ampio Pharmaceuticals
Ampio Pharmaceuticals, Inc. is a development stage biopharmaceutical company
primarily focused on the development of therapies to treat prevalent
inflammatory conditions for which there are limited treatment options. We are
developing compounds that decrease inflammation by (i) inhibiting specific
pro-inflammatory compounds by affecting specific pathways at the protein
expression and at the transcription level; (ii) activating specific
phosphatase or depletion of the available phosphate needed for the
inflammation process; and (iii) decreasing vascular permeability.
Forward Looking Statements
Ampio's statements in this press release that are not historical fact and that
relate to future plans or events are forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
Forward-looking statements can be identified by use of words such as
"believe," "expect," "plan," "anticipate," and similar expressions. These
forward-looking statements include statements regarding Ampio's expectations
with respect to the completion, timing and size of the registered direct
offering, as well as risks associated with clinical trials, expected results,
regulatory approvals, and changes in business conditions and similar events.
The risks and uncertainties involved include those detailed from time to time
in Ampio's filings with the Securities and Exchange Commission, including
without limitation, under Ampio's Annual Report on Form 10-K and Quarterly
Reports on Form 10-Q. Ampio undertakes no obligation to revise or update
these forward-looking statements, whether as a result of new information,
future events or otherwise.
Rick Giles, Director of Investor Relations, Ampio Pharmaceuticals, Inc.,
Direct: (720) 437-6530 Email: email@example.com
SOURCE Ampio Pharmaceuticals, Inc.
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