PanOptica Reports Progress with PAN-90806, a Topical Anti-VEGF Eyedrop for the Treatment of Neovascular (Wet) AMD

OIS Presentation Includes Preliminary Evidence of Safety and
Anti-VEGF Biological Activity in Phase 1/2 Trial

BERNARDSVILLE, N.J.–(BUSINESS WIRE)–PanOptica, Inc., a private biopharmaceutical company focused on
licensing and developing innovative ophthalmology therapies, today
briefed investors on the development of PAN-90806, a clinical-stage
candidate as a topical anti-vascular endothelial growth factor
(anti-VEGF) eyedrop for the treatment of neovascular eye diseases. In a
presentation at the 2015 Ophthalmology Innovation Summit at the American
Academy of Ophthalmology (OIS @ AAO) in Las Vegas, Nev., PanOptica
President and CEO Paul G. Chaney reported progress with the PAN-90806
clinical development program, including preliminary results from the
monotherapy arm of a Phase 1/2 study showing signals of safety and
anti-VEGF biological activity in patients with neovascular age-related
macular degeneration (wet AMD).

“Neovascular eye diseases frequently require continuous, life-long
treatment in order to attain optimal efficacy. A safe and effective
topical anti-VEGF eyedrop would reduce the treatment burden associated
with current therapies, potentially eliminate injection-related risks,
and would also be compatible with the growing number of combination
therapies for AMD and diabetic macular edema,” noted Elias Reichel,
M.D., Professor and Vice Chair for Research and Education in the
Department of Ophthalmology at the New England Eye Center in Boston,
Massachusetts. “The early clinical experience with PAN-90806 warrants
continued investigation of the potential benefits of this drug, which,
if proven, would allow patients and their caregivers a more active and
direct role in managing the requisite long-term therapy for their
neovascular AMD.”

Mr. Chaney presented preliminary Phase 1/2 results in which signals of
anti-VEGF biological activity were observed across all PAN-90806
monotherapy dose arms in patients with wet AMD, including at the lowest
doses. Those signals were confirmed independently by a focus panel of
retina experts. He noted that safety assessments of PAN-90806 are
ongoing, and that no treatment-related systemic adverse events (AEs)
have been reported to date; safety signals thus far have been limited to
ocular surface findings at higher doses. Mr. Chaney added that the
maximum tolerated dose (MTD) has been established for the current
formulation administered over eight weeks.

“PAN-90806 is a potent, selective, small-molecule VEGF receptor blocker
with unusually favorable characteristics for topical back-of-the-eye
delivery,” Mr. Chaney told his audience at OIS @ AAO. “Reproducible
pharmacokinetic findings demonstrate excellent target tissue
distribution to the central choroid and central retina, with
concentrations sustained at 17 hours post-dose. Moreover, topical dosing
results in extremely low systemic exposure, and animal studies suggest
that PAN-90806 performs as well as anti-VEGF antibodies administered by
intravitreal injection.”

“PAN-90806 may have utility in other chronic neovascular eye diseases,
including diabetic retinopathy and retinal vein occlusion. Researchers
have also expressed interest in studying PAN-90806 as a potential
strategy for preventing the progression to wet AMD in patients with
high-risk dry AMD,” Mr. Chaney continued. He noted that PanOptica has
initiated a Phase 1 trial of PAN-90806 in patients with proliferative
diabetic retinopathy, and that full study results from the AMD trial,
including patients in the ongoing Stage 2 of the trial treated with a
single injection of anti-VEGF therapy followed by PAN-90806 maintenance
therapy for up to three months, will be presented in 2016.

Additionally, Mr. Chaney reported that the company is developing an
advanced, next-generation formulation of PAN-90806 for use in future
trials. The new formulation appears to have strong potential for an
expanded safe and effective dose range, based on observations of
uniformly low corneal concentrations and enhanced distribution to the
central choroid and central retina.

About PAN-90806

PAN-90806 is a potent and selective inhibitor of VEGF, 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 neovascular AMD. In pre-clinical research using ocular angiogenesis
models, topically administered PAN-90806 suppressed the formation of new
blood vessels. In pharmacokinetic studies conducted in rabbits, topical
administration of PAN-90806 achieved significant and sustained levels in
the retina and choroid, 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 Novo
Ventures, Third Rock 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 clinical 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
Joe Pohl, 973-442-1555 ext.
113
Joe.pohl@smithsolve.com

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