PanOptica Reports Positive Results from Phase 1/2 Clinical Trial of PAN-90806, a Novel Topical Anti-VEGF Eye Drop

Late-Breaking Data Presented During Retina Subspecialty Day at AAO
Annual Meeting

Show Positive Biological Activity in
Patients with Wet AMD

Next-Generation Formulation Scheduled to Enter New Phase 1/2
Clinical Trial in 2017

BERNARDSVILLE, N.J.–(BUSINESS WIRE)–PanOptica, Inc., a private biopharmaceutical company focused on
licensing and developing innovative ophthalmology therapies, today
reported positive data from a Phase 1/2 study of PAN-90806, a topical
anti-vascular endothelial growth factor (anti-VEGF) eye drop in
development for the treatment of neovascular eye diseases. Based on
these results, the company plans to initiate a Phase 1/2 clinical trial
of a next-generation formulation of topical PAN-90806 in patients with
neovascular age-related macular degeneration (wet AMD) in 2017.

Scott W. Cousins, M.D., Robert Machemer Professor of Ophthalmology and
Immunology, Vice Chair for Research, and Director of the Duke Center for
Macular Diseases at Duke Eye Center, presented late-breaking data on
behalf of investigators from the Phase 1/2 study in 50 treatment-naïve
patients with wet AMD that received topical PAN-90806 during the 2016
Retina Subspecialty Day at the American Academy of Ophthalmology (AAO)
Annual Meeting in Chicago. An independent panel of retina experts that
included Dr. Cousins confirmed positive biological response to topical
PAN-90806 in approximately 45-50% of treated patients, including
outcomes such as vascular leakage, lesion morphology and vision. The
reviewers also observed signals of anti-VEGF biological activity across
all PAN-90806 monotherapy dose arms (n=40), including at the lowest
doses.

“We are encouraged by our early clinical experience with PAN-90806 and
its potential to empower patients to take a more active role in managing
chronic treatment of their AMD,” said Dr. Cousins “If proven safe and
effective through additional clinical trials, PAN-90806 topical
anti-VEGF eye drop would reduce or eliminate injection-related risks and
would be compatible with any combination therapy administered in any
way.”

No treatment-related systemic adverse events (AEs) were reported. Local
AEs were limited to ocular surface findings at the higher doses (most
commonly punctate keratopathy) that were reversible upon discontinuation
of treatment.

Dr. Cousins also reported on recent progress with the development of a
new suspension formulation of PAN-90806, which demonstrates reduced
corneal concentrations and a reduced risk of adverse corneal findings in
exploratory non-clinical pharmacokinetic and toxicology studies, while
maintaining excellent dose-dependent target tissue distribution to the
central choroid and central retina.

“Based on the clinical observations of safety and tolerability exhibited
in the first-in-man PAN-90806 Phase 1/2 study, along with the
significant progress toward a better-tolerated next generation
formulation, we plan to pursue a higher dose range in our new Phase 1/2
study for topical PAN-90806 as monotherapy next year,” stated Paul G.
Chaney, President and Chief Executive Officer, PanOptica. “PAN-90806 is
a potent, selective, small-molecule VEGF receptor blocker with unusually
favorable characteristics for topical delivery to the back-of-the-eye.
The next trial has the potential to define the optimal dose, regimen and
path toward registration of the first effective topical treatment for
wet AMD.”

PAN-90806 also is being studied in a Phase 1 trial of patients with
Proliferative Diabetic Retinopathy, and may have utility in other
chronic neovascular eye diseases, including retinal vein occlusion, and
other VEGF-dependent neovascular eye diseases. Retina specialists also
have expressed interest in studying PAN-90806 as a potential strategy
for preventing the progression to wet AMD in patients with high-risk dry
AMD.

About PAN-90806

PAN-90806 is a potent and selective inhibitor of VEGF receptor
signaling. VEGF is a protein that plays a critical role in angiogenesis
(the formation of new blood vessels) and increased permeability (leakage
from blood vessels), two pathological processes that contribute to the
vision loss associated with wet AMD. In pre-clinical research using
validated ocular angiogenesis models, topically administered PAN-90806
suppressed the formation of new blood vessels. In non-clinical
pharmacokinetic studies, topical administration of PAN-90806 achieved
significant and sustained levels in the retina and choroid of multiple
species, supporting further investigation of PAN-90806.

About PanOptica

PanOptica, Inc., is a private, venture-backed biopharmaceutical company
focused on licensing and developing a portfolio of exciting and
innovative therapeutics for major ophthalmic diseases. In April 2014,
the company closed a $45 million Series B financing backed by Third Rock
Ventures, Novo Ventures, and founding investor SV Life Sciences. The
company was co-founded by Paul Chaney, past president of OSI-Eyetech and
Martin Wax, MD, a noted academic clinician scientist working in
glaucoma, and most recently VP of research and development and head of
ophthalmology discovery and pre-clinical development at Alcon Labs, Inc.
The company seeks early-stage assets translated from other diseases and
develops select candidates through human clinical proof of concept. For
more information, please visit www.panopticapharma.com.

Contacts

SmithSolve LLC on behalf of PanOptica
Jennifer Devine, 973-442-1555
ext. 102
Jennifer.Devine@smithsolve.com