TIME-2b Study Did Not Meet the Primary Endpoint of 2-Step Reduction
in DRSS Score
Conference Call and Webcast Today, March 18th at 8:30 a.m. EDT
CINCINNATI–(BUSINESS WIRE)–Aerpio Pharmaceuticals, Inc. (Nasdaq:ARPO), a biopharmaceutical company
focused on developing compounds that activate Tie2 to treat ocular
diseases and diabetic complications, today announced top-line results
from the Company’s TIME-2b study, a Phase 2b clinical trial designed to
assess the efficacy and safety of Aerpio’s lead candidate, AKB-9778, for
patients with moderate to severe non-proliferative diabetic retinopathy
(NPDR).
Administration of AKB-9778 twice daily did not meet the study’s primary
endpoint of the percentage of patients with an improvement of two or
more steps in the study eye diabetic retinopathy severity score (DRSS)
compared to placebo. The percentage of patients achieving this endpoint
for AKB-9779 twice daily (BID) and placebo were 9.6% and 3.8%,
respectively (p=0.270). In all qualified eyes (i.e., study eyes and
fellow eyes that met the inclusion/exclusion criteria), the percentage
of eyes achieving this endpoint was 8.6% and 2.7%, for AKB-9778 BID and
placebo, respectively (p=0.158). The rates of progression to
sight-threatening complications, including diabetic macular edema (DME)
and/or proliferative diabetic retinopathy (PDR), during the 48-week
treatment period were similar between treatment groups.
AKB-9778 did show encouraging data in a number of prespecified, key
secondary endpoints, consistent with the observations in the prior Phase
2a (TIME-2) trial, including changes in Urine Albumin-Creatinine Ratio
(UACR), a measure of kidney function, and in intraocular pressure (IOP).
The company plans to advance a topical drop formulation of AKB-9778 into
clinical development and expects to initiate a Phase 1b study in the
second quarter of 2019 with results anticipated by the end of 2019.
AKB-9778 was found to be safe and well-tolerated in this patient
population through 48 weeks of twice-daily dosing. The most common
adverse events with higher incidence in the AKB-9778 BID group were
dizziness of 10.9% versus 7.0% in the placebo arm, and headache of 10.9%
compared to placebo of 3.5%. There was one death in the study, and it
was in the placebo group.
“While we are disappointed in the primary endpoint results of this
study, we are nevertheless encouraged by the fact that several other
promising findings observed in our prior 3-month Phase 2a trial have
been prospectively confirmed in this 1-year trial,” said Stephen
Hoffman, M.D., Ph.D., Chief Executive Officer of Aerpio. “We and our
clinical advisors believe that collectively these data support a
potentially important role of the Tie2 pathway for the treatment of
diabetic complications, as well as for open angle glaucoma. After a full
analysis of the study data, we plan to provide an update on the status
of the NPDR program. We would like to thank the patients and
investigators that participated in this trial.”
TIME-2b Study Design
The TIME-2b study was a double-masked, placebo-controlled, multi-center
trial designed to evaluate the effect of AKB-9778 in patients with
moderate-to-severe NPDR. 167 patients were randomized to receive 48
weeks of treatment with either AKB-9778 15 mg subcutaneously once daily
(and placebo subcutaneously once daily), AKB-9778 15 mg subcutaneously
twice daily, or placebo subcutaneously twice daily. The primary endpoint
of the TIME-2b study was the percentage of patients who improved by two
or more steps in DRSS in the study eye. One of the study’s secondary
objectives, the urine albumin to creatinine ratio or UACR, was
prospectively included based on a post-hoc analysis of this biomarker in
the TIME-2 Phase 2a clinical trial of AKB-9778 in diabetic macular edema.
Conference Call and Webcast
Aerpio management will host a live conference call and webcast at 8:30
a.m. EDT today to discuss the results from the TIME-2b study.
The live webcast and a replay may be accessed by visiting Aerpio’s
website at http://ir.aerpio.com/.
Please connect to the Company’s website at least 15 minutes prior to the
live webcast to ensure adequate time for any software download that may
be needed to access the webcast. Alternatively, please call (877)
216-7943 (U.S.) or (417) 629-5045 (international) to listen to the live
conference call. The conference ID number for the live call is 6978348.
Please dial in approximately 10 minutes prior to the call. Telephone
replay will be available approximately two hours after the call. To
access the replay, please call (855) 859-2056 (U.S.) or (404) 537-3406
(international). The conference ID number for the replay is 6978348.
About AKB-9778
AKB-9778 is being developed as a subcutaneous injection for the
treatment of non-proliferative diabetic retinopathy and as an eyedrop
formulation for the treatment of glaucoma. AKB-9778 binds to and
inhibits vascular endothelial protein tyrosine phosphatase (VE-PTP), an
important negative regulator of Tie2. Decreased Tie2 activity
contributes to vascular instability in many diseases including diabetes.
AKB-9778 activates the Tie2 receptor irrespective of extracellular
levels of its binding ligands, angiopoietin-1 (agonist) or
angiopoietin-2 (antagonist) and may be the most efficient pharmacologic
approach to maintain normal Tie2 activation.
About Aerpio Pharmaceuticals
Aerpio Pharmaceuticals, Inc. is a biopharmaceutical company focused on
advancing first-in-class compounds that activate Tie2 to treat ocular
diseases and complications of diabetes. Tie2 is an important regulator
of vascular stability and its down-regulation is found in patients with
diabetes. Down-regulation is caused by activation of two inhibitors of
Tie2, VE-PTP and Ang-2 due to hypoxia or tissue ischemia. The Company’s
lead compound, AKB-9778, is a systemically-administered small molecule
activator of the Tie2 pathway (via highly selective and potent
deactivation of VE-PTP) and is in clinical development for the treatment
of non-proliferative diabetic retinopathy. AKB-9778 is also being
investigated for its potential utility in treating diabetic nephropathy
and an eyedrop formulation is in development as a potential treatment
for open-angle glaucoma. For more information, please visit www.aerpio.com
Forward Looking Statements
This press release contains forward-looking statements. Statements in
this press release that are not purely historical are forward-looking
statements. Such forward-looking statements include, among other things,
the development of the Company’s product candidates, including AKB-9778,
the Company’s plans for future development of its product candidates,
the potential of the Tie2 pathway in treatment of diabetic
complications, and the therapeutic potential of the Company’s product
candidates. Actual results could differ from those projected in any
forward-looking statements due to several risk factors. Such factors
include, among others, the ability to raise the additional funding
needed to continue to develop AKB-9778 or other product development
plans, the inherent uncertainties associated with the drug development
process, including uncertainties in regulatory interactions, commencing
clinical trials and enrollment of patients in clinical trials,
competition in the industry in which the Company operates and overall
market conditions.
These forward-looking statements are made as of the date of this press
release, and the Company assumes no obligation to update the
forward-looking statements, or to update the reasons why actual results
could differ from those projected in the forward-looking statements,
except as required by law. Investors should consult all the information
set forth herein and should also refer to the risk factor disclosure set
forth in the reports and other documents the Company files with the SEC
available at www.sec.gov.
Contacts
Investor & Media:
Aerpio
Pharmaceuticals, Inc.
Michael Rogers
Chief Financial
Officer
mrogers@aerpio.com
or
Burns
McClellan, on behalf of Aerpio Pharmaceuticals, Inc.
Media:
Nancie
Steinberg / Robert Flamm, Ph.D.
nsteinberg@burnsmc.com
/ rflamm@burnsmc.com
or
Investors:
John
Grimaldi
jgrimaldi@burnsmc.com